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Guangping Gao, PhD

Peer-reviewed publications


Martinez-Navio JM, Fuchs SP, Pedreño-López S, Rakasz EG, Gao G, Desrosiers RC. Host anti-antibody responses following AAV-mediated delivery of antibodies against HIV and SIV in Rhesus monkeys.  Mol Ther. 2016 Feb; 24(1): 76-86.  PubMed PMID: 26444083.  PMCID: PMC4754551.

Li K, Zhang H, Qiu J, Lin Y, Liang J, Xiao X, Fu L, Wang F, Cai J, Tan Y, Zhu W, Yin W, Lu B, Xing F, Tang L, Yan M, Mai J, Li Y, Chen W, Qiu P, Su X, Gao G, Tai PW, Hu J, Yang G.  Activation of cyclic adenosine monophosphate pathway increases the sensitivity of cancer cells to the oncolytic virus M1.  Mol Ther. 2016 Feb; 24(1): 156-65.  PubMed PMID: 26373347.  PMCID: PMC4754548.

Gurda BL, De Guilhem De Lataillade A, Bell P. Zhu Y, Yu H, Wang P, Bagel J, Vite CH, Sikora T, Hinderer C, Calcedo R, Yox AD, Steet RA, Ruane T, O’Donnell P, Gao G, Wilson JM, Casal M, Ponder KP, Haskins ME.  Evaluation of AAV-mediated gene therapy for central nervous system disease in canine mucopolysaccharidosis VII.  Mol Ther. 2016 Feb; 24(2): 206-16.  PubMed PMID: 26447927.  PMCID: PMC4817811.

Ai J, Wang D, Wei Q, Li H, Gao G.  Adeno-associated virus serotype vectors efficiently transduce normal prostate tissue and prostate cancer cells.  Eur Urol. 2016 Jan; 69(1): 179-81. PubMed PMID: 26525960.  PMCID:

Schattgen SA, Gao G, Kurt-Jones, EA, Fitzgerald KA.  Cutting edge: DNA in the lung microenvironment during influenza virus infection tempers inflammation by engaging the DNA sensor AIM2.  J Immunol. 2016 Jan 1; 196(1): 29-33.  PubMed PMID: 26590313.  PMCID: PMC4793160.

Rashnonejad A, Chermahini GA, Li S, Ozkinay F, Gao G.  Large-scale production of adeno-associated viral vector serotype-9 carrying the human survival motor neuron gene.  Mol Biotechnol. 2016 Jan; 58(1): 30-6.  PubMed PMID: 26607476.  PMCID: PMC4948117.

Gessler DJ, Gao G.  Gene therapy for the treatment of neurological disorders: metabolic disorders.  Methods Mol Biol. 2016; 1382: 429-65.  PubMed PMID: 26611604.  PMCID: NIHMS799359.

Berns KI, Byrne BJ, Flotte TR, Gao G, Hauswirth WW, Herzog RW, Muzyczka N, VandenDriessche T, Xiao X, Zolotukhin S, Srivastava A.  Adeno-associated virus type 2 and hepatocellular carcinoma?  Hum Gene Ther. 2015 Dec; 26(12): 779-781.  PubMed PMID: 26690810.  PMCID: PMC4809064.

Borel F, Gernoux G, Cardozo B, Metterville JP, Toro Cabreja GC, Song L, Su Q, Gao GP, Elmallah MK, Brown RH Jr, Mueller C.  Therapeutic rAAVrh10 mediated SOD1 silencing in adult SOD1(G93A) mice and nonhuman primates.  Hum Gene Ther. 2016 Jan; 27(10): 19-31. PubMed PMID: 26710998.  PMCID: PMC4741242.

Yin H, Song C-Q, Dorkin JR, Zhu LJ, Li Y, Wu Q, Park A, Yang J, Suresh S, Bizhanova A, Gupta A, Bolukbasi MF, Walsh S, Bogorad RL, Gao G, Weng Z, Dong Y, Koteliansky V, Wolfe SA, Langer R, Xue W, Anderson DG.  Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo.  Nature Biotechnol. 2016 Mar; 34(3): 328-33. PubMed PMID: 26829318.

Kabir I, Li Z, Bui HH, Kuo MS, Gao G, Jiang XC.  Small intestine but not liver lysophospha-tidylcholine acyltransferase 3 (Lpcat3) deficiency has a dominant effect on plasma lipid metabolism.  J Biol Chem. 2016 Apr 1; 291(14): 7651-60.  PubMed PMID: 26828064. PMCID: PMC4817191.

Choudhury SR, Harris AF, Cabral DJ, Keeler AM, Sapp E, Ferreira JS, Gray-Edwards HL, Johnson        JA, Johnson AK, Su Q, Stoica L, DiFiglia M, Aronin N, Martin DR, Gao G, Sena-Esteves M. Widespread CNS gene transfer and silencing after systemic delivery of novel AAV-AS vector. Mol. Ther. 2016 Apr; 24(4): 726-35. PubMed PMID: 26708003.  PMCID: PMC4886933.

Lu Y, Ai J, Gessler D, Su Q, Tran K, Zheng Q, Xu X, Gao G.  Efficient transduction of corneal stroma by adeno-associated virus serotype vectors for implications in gene therapy of corneal diseases.  uHuHHum Gene Ther. 2016 May 16. [Epub ahead of print].  PubMed PMID: 27001051.

GuhaSarkar D, Su Q, Gao G, Sena-Esteves M.  Systemic AAV9-IFNβ gene delivery treats highly invasive glioblastoma.  Neuro-Oncol. 2016 May 18.  Pii: now097. [Epub ahead of print].  PubMed PMID: 27194146.

Ahmed SS, Schattgen SA, Frakes AE, Sikoglu EM, Su Q, Li J, Hampton TG, Denninger AR, Kirschner DA, Kaspar B, Matalon R, Gao G.  rAAV gene therapy in a Canavan’s disease mouse model reveals immune impairments and an extended pathology beyond the central nervous system.  Mol Ther. 2016 Jun; 24(6): 1030-41.  PubMed PMID: 27039844. PMCID: PMC4923332.

Fuchs SP, Martinez-Navio JM, Gao G, Desrosiers RC.  ­­­­­­­­­­­­­­­­­­­­Recombinant AAV vectors for enhanced expression of authentic IgG.  Plos One. 2016 Jun 22; 11(6):e0158009.  PubMed PMID: 27332822.  PMCID: PMC4917256.

Zhang H, Lin Y, Li K, Liang J, Xiao X, Cai J, Tan Y, Xing F, Mai J, Li Y, Chen W, Sheng L, Gu J, Zhu W, Yin W, Qiu P, Su X, Lu B, Tian X, Liu J, Lu W, Dou Y, Huang Y, Hu B, Kang Z, Gao G, Mao Z, Cheng S, Lu L, Bai X, Gong X, Yan G, Hu J.  Naturally existing oncolytic virus M1 is nonpathogenic for the non-human primates after multiple rounds of repeated intravenous injections.  Hum Gene Ther. 2016 Jun 13.  [Epub ahead of print]. PubMed: PMID: 27296553.

Shu Y, Tao Y, Wang Z, Tang Y, Li H, Dai P, Gao G, Chen ZY.  Identification of adeno-associated viral vectors (AAV) that target neonatal and adult mammalian inner ear cell subtypes.  Hum Gene Ther. 2016 Jun 24 [Epub ahead of print].  PubMed PMID: 27342665.

Guo Y, Wang D, Yang C, Qiao T, Su Q, Gao G*, Xu Z*.  A single injection of recombinant adeno-associated virus into the lumbar cistern delivers transgene expression throughout the whole spinal cord.  Mol Neurobiol 2016 Jul; 53(5): 3235-48.  *Co-corresponding authors.  PubMed PMID: 26050084; PMCID: PMC4671832.

Asokan A, Murlidharan G, Sakamoto K, Rao L, Corriher T, Wang D, Gao G, Sullivan P.  CNS restricted transduction and CRISPR/Cas9-mediated gene deletion with an engineered AAV vector.  Mol Ther. 2016, accepted.

Nagarajan A, Petersen M, Nasiri A, Butrico GM, Fung A, Ruan HB, Kursawe R, Caprio S, Thibodeau J, Bourgeois-Daigneault MC, Sun L, Gao G, Bhanot S, Jurczak M, Green M, Shulman G, Wajapeyee N.  MARCH 1 regulates insulin sensitivity by controlling surface insulin receptor levels.  Nat Comm. 2016, accepted.


Xie J, Burt D, and Gao G.  Adeno-associated virus-mediated microRNA delivery and therapeutics.  Semin Liver Dis 2015 Feb; 35(1): 81-8.  PubMed PMID: 25632938; PMCID: PMC4460833.

Ahmed S and Gao G.  Making the white matter matters: Progress in understanding Canavan's disease and therapeutic interventions through eight decades.  JIMD Rep. 2015; 19: 11-22.  PubMed PMID: 25604619.  PMCID: PMC4501231.

Gardner MR, Kattenhorn LM, Kondur HR, von Schaewen M, Dorfman T, Chiang JJ, Haworth KG, Decker JM, Alpert MD, Bailey CC, Neale ES Jr, Fellinger CH, Joshi VR, Fuchs SP, Martinez-Navio JM, Quinlan BD, Yao AY, Mouquet H, Gorman J, Zhang B, Poignard P, Nussenzweig MC, Burton DR, Kwong PD, Piatak M Jr, Lifson JD, Gao G, Desrosier RC, Evans DT, Hahn BH, Ploss A, Cannon PM, Seaman MS, and Farzan M.  AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges.  Nature 2015 Mar 5; 519(7541): 87-91.  PubMed PMID: 25707797. PMCID: PMC4352131.

Gruntman AM, Su L, Su Q, Gao G, Mueller C, Flotte TR.  Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.  Hum Gene Ther Methods 2015 Apr; 26(2): 71-6.  PubMed PMID: 25819833.  PMCID: PMC4403013.

Zhao-Shea R, DeGroot S, Liu L, Vallaster M, Pang X, Su Q, Gao G, Rando O, Martin G, George O, Gardner P, and Tapper A.  Increased CRF signaling in a ventral tegmental area-interpeduncular nucleus-medial habenula circuit induces anxiety during nicotine withdrawal.  Nat Commun 2015 Apr 21; 6: 6770. PubMed PMID: 25898242.  PMCID: PMC4405813.  Erratum in: Nat Commun. 2015 Jul 2; 6: 7625.  PubMed PMID: 26133887. 

Prendiville TW, Guo H, Lin Z, Zhou P, Stevens SM, He A, VanDusen N, Chen J, Zhong L, Wang DZ, Gao G, Pu WT.  Novel roles of GATA4/6 in the postnatal heart identified through temporally controlled, cardiomyocyte-specific gene inactivation by adeno-associated virus delivery of Cre recombinase.  PLoS One 2015 May 29; 10(5):e0128105.  PubMed PMID: 26023924.  PMCID: PMC4449121. 

He X, Xie J, Zhang D, Su Q, Sai X, Bai R, Chen C, Luo X, Gao G*, and Pan W*.  rAAV8-mediated inhibition of miRNA-21 protects mice against the lethal schistosome infection by repressing both IL-13 and TGF-β1 pathways.  *Co-corresponding authors. Hepatology 2015 Jun; 61(6): 2008-17.  PubMed PMID: 25546547; PMCID: PMC4441614.

Ngolab J, Liu L, Zhao-Shea R, Gao G, Gardner P, and Tapper A. Functional upregulation of α4* nicotinic acetylcholine receptors in VTA GABAergic neurons increases sensitivity to nicotine reward. J Neurosci 2015 Jun 3; 35(22): 8570-8.  PubMed PMID: 26041923.  PMCID: PMC4588598.

Wang D, Mou H, Li S, Li Y, Hough S, Tran K, Li J, Yin H, Anderson DG, Sontheimer EJ, Weng Z*, Gao G*, Xue W*. Adenovirus-mediated somatic genome editing of Pten by CRISPR/Cas9 in mouse liver in spite of Cas9-specific immune responses. Hum Gene Ther 2015 Jul; 26(7): 432-42.  *Co-corresponding authors.  PubMed PMID: 26086867.  PMCID: PMC4509492.

Li L, He ZY, Wei XW, Gao G, and Wei YQ. Challenges in CRISPR/CAS9 delivery: Potential roles of nonviral vectors. Hum Gene Ther. 2015 July; 26(7): 452-462.  PubMed PMID: 26176432.

Pépin D, Sosulski A, Zhang L, Wang D, Vathipadiekal V, Hendren K, Coletti CM, Yu A, Castro CM, Birrer, MJ, Gao G, and Donahoe PK.  AAV9 delivering a modified human Mullerian inhibiting substance as a gene therapy in patient-derived xenografts of ovarian cancer.  Proc Natl Acad Sci U S A. 2015 Aug 11; 112(32): E4418-27. PubMed PMID: 26216943.  PMCID: PMC4538667.

Weismann CM, Ferreira J, Keeler AM, Su Q, Qui L, Shaffer SA, Xu Z, Gao G, Sena-Esteves M.  Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan.  Hum Mol Genet 2015 Aug 1; 24(15): 4353-64.  PubMed PMID: 25964428.  PMCID: PMC4492398.

Fuchs SP, Martinez-Navio JM, Piatak Jr MP, Lifson JD, Gao G, Desrosiers RC.  AAV-delivered antibody mediates significant protective effects against SIVmac239 challenge in the absence of neutralizing activity.  PLoS Pathog 2015 Aug 6; 11(8):e1005090. PubMed PMID: 26248318.  PMCID: PMC4527674.

Cahoon JM, Rai RR, Carroll LS, Uehara H, Zhang X, O’Neil CL, Medina RJ, Das SK, Muddana SK, Olson PR, Nielson S, Walker K, Flood MM, Messenger WB, Archer BJ, Barabas P, Krizaj D, Gibson CC, Li DY, Koh GY, Gao G, Stitt AW, Ambati BK.  Intravitreal AAV2.COMP-Ang1 prevents neurovascular degeneration in a murine model of diabetic retinopathy.  Diabetes 2015 Dec; 64(12): 4247-49.  PubMed PMID: 26340930.  PMCID: PMC4657578.

Li S, Ling C, Zhong L, Li M, Su Q, He R, Tang Q, Greiner DL, Shultz LD, Brehm MA, Flotte TR, Mueller C, Srivastava A*, Gao G*.  Efficient and targeted transduction of nonhuman primate liver with systemically optimized AAV3B vectors. *Co-corresponding authors. Mol Ther. 2015 Dec. 23(12): 1867-1876.  PubMed PMID: 26403887. PMCID: PMC4700112.


Pang X, Hogan EM, Casserly A, Gao G, Gardner PD, and Tapper AR. Dicer expression is essential for adult midbrain dopaminergic neuron maintenance and survival. Mol Cell Neurosci. 2014 Jan; 58: 22-8. PubMed PMID: 24184162; PMCID: PMC3944994.

Wang H, Yang B, Qiu L, Yang C, Kramer J, Su Q, Guo Y, Brown RH Jr, Gao G*, and Xu Z*. Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis. Hum Mol Genet. 2014 Feb 1; 23(3): 668-81. * Co-corresponding Authors.  PubMed PMID: 24108104; PMCID: PMC3888258.

Liu L, Nam M, Fan W, Akie TE, Hoaglin DC, Gao G, Keaney Jr JF and Cooper MP. Nutrient sensing by the mitochondrial transcription machinery dictates oxidative phosphorylation.  J Clin Invest. 2014 Feb; 124(2):768-84.  PubMed PMID: 24430182; PMCID: PMC4381729.

Gao K, Li M, Zhong L, Su Q, Li J, Li S, He R, Zhang Y, Hendricks G, Wang J, and Gao G. Empty virions in AAV8 vector preparations reduce transduction efficiency and may cause total viral particle dose-limiting side-effects. Mol Ther Methods Clin Dev. 2014 1(9):20139. PubMed PMID: 25485285; PMCID: PMC4255953.

Wang D, Zhong L, Nahid MA, and Gao G.  The potential of adeno-associated viral vectors for gene delivery to muscle tissue.  Expert Opin Drug Deliv. 2014 Mar, 11(3):345-64.  PubMed PMID: 24386892; PMCID: PMC4098646.

Xu H, Zhang L, Gu L, Lu L, Gao G, Li W, Xu G, Wang J, Gao F, Xu JY, Yao J, Wang F, Zhang J, Xu GT. Subretinal delivery of AAV2-mediated human erythropoietin gene is protective and safe in experimental diabetic retinopathy.  Invest Ophthalmol Vis Sci. 2014 Mar 13: 55(3): 1519-30. PubMed PMID: 24508793.

Yang B, Li S, Wang H, Guo Y, Gessler DJ, Cao C, Su Q, Kramer J, Zhong L, Ahmed SS, Zhang H, He R, Desrosiers RC, Brown R, Xu Z, and Gao G.  Global CNS transduction of adult mice by intravenously delivered rAAVrh8 and rAAVrh10 and nonhuman primates by rAAVrh10.  Mol Ther. 2014 Jul; 22(7): 1299-309.  PubMed PMID: 24781136; PMCID: PMC4089005.

Wang D and Gao G. State-of-the-art human gene therapy: Part I. Gene delivery technologies. Discov Med. 2014 Jul-Aug;18(97):67-77.  PubMed PMID: 25091489; PMCID: PMC4440413.

Vernia S, Cavanagh-Kyros J, Garcia-Haro L, Sabio G, Barrett T, Young Jung D, Kim JK, Xu J, Shulha HP, Garber M, Gao G, and Davis RJ. The PPARa-FGF21 hormone axis contributes to metabolic regulation by the hepatic JNK signaling pathway. Cell Metab 2014 Sep 2; 20(3):512-25.  PubMed PMID: 25043817; PMCID: PMC4156535.

Vincent M, Gao G, and Jacobson L.  Comparison of the efficacy of five adeno-associated virus vectors for transducing dorsal raphé nucleus cells in the mouse. J Neurosci Methods. 2014 Sep 30; 235:189-92. PubMed PMID: 25046366; PMCID: PMC4150843.

Wang D and Gao G. State-of-the-art human gene therapy: Part II: Gene therapy strategies and clinical applications.  Discov Med 2014 Sep; 18(98):151-61.  PubMed PMID: 25227756; PMCID: PMC4440458.

Ayuso E, Blouin V, Lock M, McGorray, S, Leon X, Alvira MR, Auricchio A, Bucher S, Chtarto A, Clark KR, Darmon C, Doria M, Fountain W, Gao G, Gao K, Giacca M, Kleinschmidt J, Leuchs B, Melas C, Mizukami H, Müller M, Noordman Y, Bockstael O, Ozawa K, Pythoud C, Sumaroka M, Surosky R, Tenenbaum L, van der Linden I, Weins B, Wright JF, Zhang X, Zentilin L, Bosch F, Snyder RO, and Moullier P.  Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.  Human Gene Therapy 2014 Nov 25: 977-987.  PubMed PMID: 25275822; PMCID: PMC4236062.

Ahmed SS and Gao G. Examination of the blood brain barrier integrity in a mouse model of the neurodegenerative Canavan’s disease.  J Neurol Disord 2014 Nov, 2(6). pii: i105. PubMed PMID: 26550581.  PMCID: PMC4635683.


van der Born IM, Moser RP, Gao G, Mondo E, O’Connell D, Gounis MJ, McGowan S, Chaurette J, Bishop N, Sena-Esteves MS, Mueller C, and Aronin N.  Finding the striatum in sheep: use of a multi-modal guided approach for convection enhanced delivery. J Huntingtons Dis. 2013; 2(1):41-5. PubMed PMID: 25063428.

van der Born IM, Moser RP, Gao G, Sena-Esteves M, Aronin N, and Gounis MJ.  Frameless multimodal image guidance of localized convection-enhanced delivery of therapeutics in the brain.  J Neurointerv Surg. 2013 Jan 1;5(91):69-72.  PubMed PMID: 22193239; PMCID: PMC3533401.

Sun L, Tu L, Gao G, Sun X, Duan J and Lu Y.  Assessment of a passive immunity mouse model to quantitatively analyze the impact of neutralizing antibodies on adeno-associated virus-mediated gene transfer.  J Immunol Methods. 2013 Jan 31; 387(1-2): 114-20.  PubMed PMID: 23063691.

Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanova AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, and Gao G.  Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.  Mol Ther. 2013 Feb; 21(2): 266-8. PubMed PMID: 23369965; PMCID: PMC3594019.

Velez G, Weingarden AR, Lei H, Kazlauskas A, and Gao G. SU9518 inhibits proliferative vitreoretinopathy in fibroblast and genetically modified Müller cell–induced rabbit models. Invest Ophthalmol Vis Sci. 2013 Feb 19; 54(2):1392-7. PubMed PMID: 23341018; PMCID: PMC3597189.

Gruntman AM, Bish LT, Mueller C, Sweeney HL, Flotte TR and Gao G. Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14: Unit 14D.3. PubMed PMID: 23408131; PMCID: PMC3641885.

Luo L, Zhang X, Hirano Y, Tyagi P, Barabás P, Uehara H, Miya TR, Singh N, Archer B, Qazi Y, Jackman K, Das SK, Olsen T, Chennamaneni SR, Stagg BC, Ahmed F, Emerson L, Zygmunt K, Whitaker R, Mamalis C, Huang W, Gao G, Srinivas SP, Krizaj D, Baffi J, Ambati J, Kompella UB, and Ambati BK. Targeted intraceptor nanoparticle therapy reduces angiogenesis and fibrosis in primate and murine macular degeneration. ACS Nano. 2013 Apr 23; 7(4): 3264-75. PubMed PMID: 23464925; PMCID: PMC3634882.

Venkatesh A, Ma S, Langellotto F, Gao G* and Punzo C*. Retinal gene delivery by rAAV and DNA electroporation. Curr Protoc Microbiol. 2013; Chapter 14: Unit 14D.4.  PubMed PMID: 23408132; PMCID: PMC3643812. * Co-corresponding authors

Ahmed SS, and Gao G. Gene therapy for Canavan's disease takes a step forward. Mol Ther. 2013 Mar; 21(3): 505-6. PubMed PMID: 23449107; PMCID: PMC3589154.

Zhong L, Malani N, Li M, Brady T, Xie J, Bell P, Li S, Jones H, Wilson JM, Flotte TR, Bushman FD* and Gao G*. Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction. Hum Gene Ther. 2013 May; 24(5): 520-5.  PubMed PMID: 23621841; PMCID: PMC3655627.

Amir M, Zhao E, Fontana L, Rosenberg H, Tanaka K, Gao G, Czaja MJ. Inhibition of hepatocyte autophagy increases tumor necrosis factor-dependent liver injury by promoting caspase-8 activation. Cell Death Differ. 2013 Jul; 20(7):878-87. PubMed PMID: 23519075; PMCID: PMC3679456.

Ahmed SS, Li J, Godwin J, Gao G* and Zhong L*. Gene transfer in the liver using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14: Unit 14D.6. doi: 10.1002/9780471729259.mc14d06s29. PubMed PMID: 23686826; PMCID: PMC4161158. *Co-corresponding authors.

Zhong L, Li S, Li M, Xie J, Zhang Y, Lee B, Batshaw ML, Wilson JM, and Gao G. Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer. Human Gene Ther. 2013 Sep; 24(9): 814-9.  PubMed PMID: 24010702; PMCID: PMC3768231.

Jakovcevski M, Bharadwaj R,  Straubhaar J, Gao G, Gavin DP, Jakovcevski I, Mitchell AC, and Akbarian S. Prefrontal cortical dysfunction after overexpression of histone deacetylase 1. Biol Psychiatry. 2013 Nov 1; 74(9): 696-705. PubMed PMID: 23664640; PMCID: PMC3797203.

Ahmed SS, Li H, Cao C, Sikoglu EM, Denninger AR, Su Q, Eaton S, Liso Navarro AA, Xie J, Szucs S, Zhang H, Moore C, Kirschner D, Seyfried TN, Flotte TR, Matalon R, and Gao G.  A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS gene therapy in Canavan mice. Mol Ther. 2013 Dec; 21(12); 2136-47. PubMed PMID: 23817205; PMCID: PMC3863789.

Stoica L, Ahmed SS, Gao G* and Sena-Esteves M*.  Gene transfer to the CNS using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14: Unit 14D.5. PubMed PMID: 23686825; PMCID: PMC4135440. * Co-corresponding authors.

Song L, Li X, Jayandharan GR, Wang Y, Aslanidi GV, Ling C, Zhong L, Gao G, Yoder MC, Ling C, Tan M, and Srivastava A. High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivo. PLoS One. 2013; 8(3): e58757. PubMed PMID: 23516552; PMCID: PMC3597592.

Luo L, Uehara H, Zhang X, Das SD, Olsen T, Holt D, Simonis JM, Jackman K, Singh N, Miya TR, Huang W, Ahmed F, Bastos-Carvalho A, Le YZ, Mamalis C, Chiodo VA, Hauswirth WW, Baffi J, Lacal PM, Orecchia A, Ferrara N, Gao G, Young-hee K, Fu Y, Owen L, Albuquerque R, Baehr W, Thomas K, Li DY, Chalam KV, Shibuya M, Grisanti S, Wilson DJ, Ambati J, Ambati BK. Photoreceptor avascular privilege is shielded by soluble VEGF receptor-1.  Elife 2013; Jun 18;2: e00324. doi: 10.7554/eLife.00324. Print 2013.  PubMed PMID: 23795287; PMCID: PMC3687373.


Xie J, Xie Q, Zhang H, Ameres SL, Hung JH, Su Q, He R, Mu X, Seher Ahmed S, Park S, Kato H, Li C, Mueller C, Mello CC, Weng Z, Flotte TR, Zamore PD, and Gao G. MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. Mol Ther. 2011 Mar; 19(3): 526-35. PubMed PMID: 21179009; PMCID: PMC3048189.

Lu J, Wang Q, Wang H, Li G, and Gao G. Molecular characterization of adenoviruses in fecal samples of captively bred rhesus macaques in China. Vet Microbiol. 2011 May 5; 149(3-4): 461-6. PubMed PMID: 21215533.

Bell CL, Vandenberghe LH, Bell P, Limberis MP, Gao GP, Van Vliet K, Agbandje-McKenna M, and Wilson JM.  The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice.  J Clin Invest. 2011 Jun; 121(6): 2427-35.  PubMed PMID: 21576824.  PMCID: PMC3104778.

Bell P*, Gao G*, Haskins ME, Wang L, Sleeper M, Wang H, Cacedo R, Vandenberghe LH, Chen SJ, Weisse C, Withnall E, and Wilson JM. Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs. Hum Gene Ther. 2011 Aug; 22(8): 985-97.  PubMed PMID: 21204705; PMCID: PMC3159528. *Equal contribution first authors.

Bish LT, Sleeper MM, Reynolds C, Gazzara J, Withnall E, Singletary GE, Buchlis G, Hui D, High KA, Gao G, Wilson JM, and Sweeney HL.  Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines.  Hum Gene Ther. 2011 Aug; 22(8): 969-77. PubMed PMID: 21542669; PMCID: PMC3159526.

Gao G, Bish LT, Sleeper MM, Mu X, Sun L, Lu Y, Duan J, Hu C, Wang L, and Sweeney HL.  Transendocardial delivery of AAV6 results in highly efficient and global cardiac gene transfer in rhesus macaques.  Hum Gene Ther. 2011 Aug; 22(8): 979-84.  PubMed PMID: 21563985.

Zhang H, Yang B, Mu X, Ahmed SS, Su Q, He R, Wang H, Mueller C, Sena-Esteves M, Brown R, Xu Z, and Gao G.  Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system.  Mol Ther. 2011 Aug; 19(8): 1440-8. PubMed PMID: 21610699; PMCID: PMC3149178.

Gao G, Zhong L, and Danos O.  Exploiting natural diversity of AAV for the design of vectors with novel properties.  Methods Mol Biol. 2011; 807: 93-118.  PubMed PMID: 22034027.

Bell P, Wang L, Gao G, Haskins ME, Tarantal AF, McCarter RJ, Zhu Y, Yu H, and Wilson JM.  Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates.  Mol Genet Metab. 2011 Nov; 104(3): 395-403.  PubMed PMID: 21778099; PMCID: PMC3269907.

Xie J, Ameres SL, Friedline R, Hung JH, Zhang Y, Xie Q, Zhong L, Su Q, He R, Li M, Li H, Mu X, Zhang H, Broderick JA, Kim JK, Weng Z, Flotte TR, Zamore PD, and Gao G.  Long-term, efficient inhibition of microRNA function in mice using rAAV vectors.  Nat Methods. 2012 Mar 4; 9(4): 403-9.  PubMed PMID: 22388288; PMCID: PMC3420816.

Beckett T, Bonneau L, Howard A, Blanchard J, Borda J, Weiner DJ, Wang L, Gao GP, Kolls JK, Bohm R, Liggitt D, and Weiss DJ.  Inhalation of nebulized perfluorochemical enhances recombinant adenovirus and adeno-associated virus-mediated gene expression in lung epithelium.  Hum Gene Ther Methods. 2012 Apr; 23(2): 98-110. PubMed PMID: 22568624; PMCID: PMC4015222.

Mueller C, Zhong L, Ratner D, Esteves-Sena M, and Gao G. Production and discovery of novel recombinant adeno-associated virus vectors. Curr Protoc Microbiol. 2012 Aug; Chapter 14: Unit 14D.1. PubMed PMID: 22875565; PMCID: PMC4358842.  

Gruntman AM, Mueller C, Flotte TR, and Gao G.  Gene transfer in the lung using recombinant adeno-associated virus. Curr Protoc Microbiol. 2012 Aug; Chapter 14: Unit14D.2. PubMed PMID: 22875566; PMCID: PMC3486738.


Maguire CA, Gianni D, Meijer DH, Shaket LA, Wakimoto H, Rabkin SD, Gao G, and Sena-Esteves M. Directed evolution of adeno-associated virus for glioma cell transduction. J Neurooncol. 2010 Feb; 96(3): 337-47. PubMed PMID: 19618115; PMCID: PMC2892971.

Jakovcevski M, Guo Y, Su Q, Gao G, and Akbarian S. rAAV9--a human-derived adeno-associated virus vector for efficient transgene expression in mouse cingulate cortex. Cold Spring Harb Protoc. 2010 Apr; 2010(4): pdb.prot5417. PubMed PMID: 20360371.

Sun X, Lu Y, Bish LT, Calcedo R, Wilson JM, and Gao G. Molecular analysis of vector genome structures after liver transduction by conventional and self-complementary adeno-associated viral serotype vectors in murine and nonhuman primate models. Hum Gene Ther. 2010 Jun; 21(6): 750-61. PubMed PMID: 20113166; PMCID: PMC2938357.

Goodman AL, Epp C, Moss D, Holder AA, Wilson JM, Gao GP, Long CA, Remarque EJ, Thomas AW, Ammendola V, Colloca S, Dicks MD, Biswas S, Seibel D, van Duivenvoorde LM, Gilbert SC, Hill AV, and Draper SJ. New candidate vaccines against blood-stage Plasmodium falciparum malaria: prime-boost immunization regimens incorporating human and simian adenoviral vectors and poxviral vectors expressing an optimized antigen based on merozoite surface protein 1. Infect Immun. 2010 Nov; 78(11): 4601-12. PubMed PMID: 20713623; PMCID: PMC2976338.

Hendricks G and Gao G. Transmission electron tomography of adeno-associated virus (AAV).  Microscopy and Microanalysis 2010, 16 (Suppl. 2), pp 1102-1103. doi:10.1017/S1431927610055716.


Calcedo R, Vandenberghe LH, Gao G, Lin J, and Wilson JM. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis. 2009 Feb 1; 199(3): 381-90. PubMed PMID: 19133809.

Zhang Z, Zheng Q, Han J, Gao G, Liu J, Gong T, Gu Z, Huang Y, Sun X, and He Q. The targeting of 14-succinate triptolide-lysozyme conjugate to proximal renal tubular epithelial cells. Biomaterials. 2009 Mar; 30(7): 1372-81. PubMed PMID: 19100618.

Vandenberghe LH, Wilson JM, and Gao G. Tailoring the AAV vector capsid for gene therapy. Gene Ther. 2009 Mar; 16(3): 311-9. PubMed PMID: 19052631.

Pichla-Gollon SL, Lin SW, Hensley SE, Lasaro MO, Herkenhoff-Haut L, Drinker M, Tatsis N, Gao GP, Wilson JM, Ertl HC, and Bergelson JM. Effect of preexisting immunity on an adenovirus vaccine vector: in vitro neutralization assays fail to predict inhibition by antiviral antibody in vivo. J Virol. 2009 Jun; 83(11): 5567-73. PubMed PMID: 19279092; PMCID: PMC2681979.

Gao G, Wang Q, Calcedo R, Mays L, Bell P, Wang L, Vandenberghe LH, Grant R, Sanmiguel J, Furth EE, and Wilson JM. Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. Hum Gene Ther. 2009 Sep; 20(9): 930-42. PubMed PMID: 19441963; PMCID: PMC2829282.

Zhang H, Xie J, Xie Q, Wilson JM, and Gao G. Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production. Hum Gene Ther. 2009 Sep; 20(9): 922-9. PubMed PMID: 19618999.

Vandenberghe LH, Breous E, Nam HJ, Gao G, Xiao R, Sandhu A, Johnston J, Debyser Z, Agbandje-McKenna M, and Wilson JM. Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints. Gene Ther. 2009 Dec; 16(12): 1416-28. PubMed PMID: 19727141; PMCID: PMC2795093.


Reyes-Sandoval A, Sridhar S, Berthoud T, Moore AC, Harty JT, Gilbert SC, Gao G, Ertl HC, Wilson JC, and Hill AV. Single-dose immunogenicity and protective efficacy of simian adenoviral vectors against Plasmodium berghei. Eur J Immunol. 2008 Mar; 38(3): 732-41. PubMed PMID: 18266272.

Li D, Guang W, Abuzeid WM, Roy S, Gao GP, Sauk JJ, and O'Malley BW Jr. Novel adenoviral gene delivery system targeted against head and neck cancer. Laryngoscope. 2008 Apr; 118(4): 650-8. PubMed PMID: 18176343.

Sridhar S, Reyes-Sandoval A, Draper SJ, Moore AC, Gilbert SC, Gao GP, Wilson JM, and Hill AV. Single-dose protection against Plasmodium berghei by a simian adenovirus vector using a human cytomegalovirus promoter containing intron A. J Virol. 2008 Apr; 82(8): 3822-33. PubMed PMID: 18256155; PMCID: PMC2293012.

Gao G, Wang Q, Dai Z, Calcedo R, Sun X, Li G, and Wilson JM. Adenovirus-based vaccines generate cytotoxic T lymphocytes to epitopes of NS1 from dengue virus that are present in all major serotypes. Hum Gene Ther. 2008 Sep; 19(9): 927-36. PubMed PMID: 18788905.

Bish LT, Sleeper MM, Brainard B, Cole S, Russell N, Withnall E, Arndt J, Reynolds C, Davison E, Sanmiguel J, Wu D, Gao G, Wilson JM, and Sweeney HL. Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 achieves global cardiac gene transfer in canines. Mol Ther. 2008 Dec; 16(12): 1953-9. PubMed PMID: 18813281; PMCID: PMC3241935.

Bish LT, Morine K, Sleeper MM, Sanmiguel J, Wu D, Gao G, Wilson JM, and Sweeney HL. Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat. Hum Gene Ther. 2008 Dec; 19(12): 1359-68. PubMed PMID: 18795839; PubMed Central PMCID: PMC2940566. 


Pichla-Gollon SL, Drinker M, Zhou X, Xue F, Rux JJ, Gao GP, Wilson JM, Ertl HC, Burnett RM, and Bergelson JM. Structure-based identification of a major neutralizing site in an adenovirus hexon. J Virol. 2007 Feb; 81(4): 1680-9. PubMed PMID: 17108028; PMCID: PMC1797575.

Tatsis N, Blejer A, Lasaro MO, Hensley SE, Cun A, Tesema L, Li Y, Gao GP, Xiang ZQ, Zhou D, Wilson JM, and Ertl HC. A CD46-binding chimpanzee adenovirus vector as a vaccine carrier. Mol Ther. 2007 Mar; 15(3): 608-17. PubMed PMID: 17228314.

100. Kobinger GP, Figueredo JM, Rowe T, Zhi Y, Gao G, Sanmiguel JC, Bell P, Wivel NA, Zitzow LA, Flieder DB, Hogan RJ, and Wilson JM. Adenovirus-based vaccine prevents pneumonia in ferrets challenged with the SARS coronavirus and stimulates robust immune responses in macaques. Vaccine. 2007 Jul 9; 25(28): 5220-31. PubMed PMID: 17559989.


De BP, Heguy A, Hackett NR, Ferris B, Leopold PL, Lee J, Pierre L, Gao G, Wilson JM, and Crystal RG. High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses. Mol Ther. 2006 Jan; 13(1): 67-76. PubMed PMID: 16260185.

Gao G, Lu Y, Caceldo R, Grant RL, Bell P, Wang L, Figueredo J, Lock M, and Wilson JM:  Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates.  Mol Ther. 2006 Jan; 13(1): 77-87.  Erratum in: Mol Ther. 2006 Jul; 14(1):150.  PubMed PMID: 16219492.

Tatsis N, Tesema L, Robinson ER, Giles-Davis W, McCoy K, Gao GP, Wilson JM, and Ertl HC. Chimpanzee-origin adenovirus vectors as vaccine carriers. Gene Ther. 2006 Mar; 13(5): 421-9. PubMed PMID: 16319951.

Kobinger GP, Feldmann H, Zhi Y, Schumer G, Gao G, Feldmann F, Jones S, and Wilson JM. Chimpanzee adenovirus vaccine protects against Zaire Ebola virus. Virology. 2006 Mar 15; 346(2): 394-401. PubMed PMID: 16356525.

Zhi Y, Figueredo J, Kobinger GP, Hagan H, Calcedo R, Miller JR, Gao G, and Wilson JM. Efficacy of severe acute respiratory syndrome vaccine based on a nonhuman primate adenovirus in the presence of immunity against human adenovirus. Hum Gene Ther. 2006 May; 17(5): 500-6. PubMed PMID: 16716107.

Gao GP, Lu Y, Sun X, Johnston J, Calcedo R, Grant R, and Wilson JM. High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes. J Virol. 2006 Jun; 80(12): 6192-4. PubMed PMID: 16731960; PMCID: PMC1472562.

Bell P, Moscioni AD, McCarter RJ, Wu D, Gao G, Hoang A, Sanmiguel JC, Sun X, Wivel NA, Raper SE, Furth EE, Batshaw ML, and Wilson JM. Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. Mol Ther. 2006 Jul; 14(1): 34-44. PubMed PMID: 16682254.

Moscioni D, Morizono H, McCarter RJ, Stern A, Cabrera-Luque J, Hoang A, Sanmiguel J, Wu D, Bell P, Gao GP, Raper SE, Wilson JM, and Batshaw ML. Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors. Mol Ther. 2006 Jul; 14(1): 25-33. PubMed PMID: 16677864.

Kitajima K, Marchadier DH, Miller GC, Gao GP, Wilson JM, and Rader DJ. Complete prevention of atherosclerosis in apoE-deficient mice by hepatic human apoE gene transfer with adeno-associated virus serotypes 7 and 8. Arterioscler Thromb Vasc Biol. 2006 Aug; 26(8): 1852-7. PubMed PMID: 16763161.

Kelkar S, De BP, Gao G, Wilson JM, Crystal RG, and Leopold PL. A common mechanism for cytoplasmic dynein-dependent microtubule binding shared among adeno-associated virus and adenovirus serotypes. J Virol. 2006 Aug; 80(15): 7781-5. PubMed PMID: 16840360; PMCID: PMC1563724.

Vandenberghe LH, Wang L, Somanathan S, Zhi Y, Figueredo J, Calcedo R, Sanmiguel J, Desai RA, Chen CS, Johnston J, Grant RL, Gao G, and Wilson JM. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med. 2006 Aug; 12(8): 967-71. PubMed PMID: 16845388.


Zhang H, Gao G, Clayburne G, and Schumacher HR. Elimination of rheumatoid synovium in situ using a Fas ligand 'gene scalpel'. Arthritis Res Ther. 2005; 7(6): R1235-43. PubMed PMID: 16277676; PMCID: PMC1297566.

Rivera VM, Gao GP, Grant RL, Schnell MA, Zoltick PW, Rozamus LW, Clackson T, and Wilson JM. Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood. 2005 Feb 15; 105(4): 1424-30. PubMed PMID: 15507527.

Shifrin AL, Chirmule N, Gao GP, Wilson JM, and Raper SE. Innate immune responses to adenoviral vector-mediated acute pancreatitis. Pancreas. 2005 Mar; 30(2): 122-9. PubMed PMID: 15714134.

Varnavski AN, Calcedo R, Bove M, Gao G, and Wilson JM. Evaluation of toxicity from high-dose systemic administration of recombinant adenovirus vector in vector-naive and pre-immunized mice. Gene Ther. 2005 Mar; 12(5): 427-36. PubMed PMID: 15647774.

Jennings K, Miyamae T, Traister R, Marinov A, Katakura S, Sowders D, Trapnell B, Wilson JM, Gao G, and Hirsch R. Proteasome inhibition enhances AAV-mediated transgene expression in human synoviocytes in vitro and in vivo. Mol Ther. 2005 Apr; 11(4): 600-7. PubMed PMID: 15771962.

Zhi Y, Kobinger GP, Jordan H, Suchma K, Weiss SR, Shen H, Schumer G, Gao G, Boyer JL, Crystal RG, and Wilson JM. Identification of murine CD8 T cell epitopes in codon-optimized SARS-associated coronavirus spike protein. Virology. 2005 Apr 25; 335(1): 34-45. PubMed PMID: 15823604.

Lai M, Wang F, Rohan JG, Maeno-Hikichi Y, Chen Y, Zhou Y, Gao G, Sather WA, and Zhang JF.  A tctex1-Ca2+ channel complex for selective surface expression of Ca2+ channels in neurons.  Nat Neurosci. 2005 Apr; 8(4): 435-42. PubMed PMID:15768038.

Deriy L, Ogawa H, Gao GP, and Galili U. In vivo targeting of vaccinating tumor cells to antigen-presenting cells by a gene therapy method with adenovirus containing the alpha1,3galactosyltransferase gene. Cancer Gene Ther. 2005 Jun; 12(6): 528-39. PubMed PMID: 15818383.

Gao G, Vandenberghe LH, and Wilson JM. New recombinant serotypes of AAV vectors. Curr Gene Ther. 2005 Jun; 5(3): 285-97. PubMed PMID: 15975006.

Bell P, Limberis M, Gao G, Wu D, Bove MS, Sanmiguel JC, and Wilson JM. An optimized protocol for detection of E coli beta-galactosidase in lung tissue following gene transfer. Histochem Cell Biol. 2005 Jul; 124(1): 77-85. PubMed PMID: 15947941.

Bell P, Wang L, Lebherz C, Flieder DB, Bove MS, Wu D, Gao GP, Wilson JM, and Wivel NA. No evidence for tumorigenesis of AAV vectors in a large-scale study in mice. Mol Ther. 2005 Aug; 12(2): 299-306. PubMed PMID: 16043099.

Li Z, Qiao H, Lebherz C, Choi SR, Zhou X, Gao G, Kung HF, Rader DJ, Wilson JM, Glickson JD, and Zhou R. Creatine kinase, a magnetic resonance-detectable marker gene for quantification of liver-directed gene transfer. Hum Gene Ther. 2005 Dec; 16(12): 1429-38. PubMed PMID: 16390274.


Katakura S, Jennings K, Watanabe S, Adachi E, Thornton S, Gao GP, Wilson JM, Burstein H, Trapnell B, and Hirsch R. Recombinant adeno-associated virus preferentially transduces human, compared to mouse, synovium: implications for arthritis therapy. Mod Rheumatol. 2004; 14(1): 18-24. PubMed PMID: 17028800.

Pinto AR, Fitzgerald JC, Gao GP, Wilson JM, and Ertl HC. Induction of CD8+ T cells to an HIV-1 antigen upon oral immunization of mice with a simian E1-deleted adenoviral vector. Vaccine. 2004 Jan 26; 22(5-6): 697-703. PubMed PMID: 14741162.

Ziegler RJ, Lonning SM, Armentano D, Li C, Souza DW, Cherry M, Ford C, Barbon CM, Desnick RJ, Gao G, Wilson JM, Peluso R, Godwin S, Carter BJ, Gregory RJ, Wadsworth SC, and Cheng SH. AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice. Mol Ther. 2004 Feb; 9(2): 231-40. PubMed PMID: 14759807.

Sarkar R, Tetreault R, Gao G, Wang L, Bell P, Chandler R, Wilson JM, and Kazazian HH Jr. Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. Blood. 2004 Feb 15; 103(4): 1253-60. PubMed PMID: 14551134.

Gao G, Lebherz C, Weiner DJ, Grant R, Calcedo R, McCullough B, Bagg A, Zhang Y, and Wilson JM. Erythropoietin gene therapy leads to autoimmune anemia in macaques. Blood. 2004 May 1; 103(9): 3300-2. PubMed PMID: 14695227.

Roy S*, Gao G*, Lu Y, Zhou X, Lock M, Calcedo R, and Wilson JM. Characterization of a family of chimpanzee adenoviruses and development of molecular clones for gene transfer vectors. Hum Gene Ther. 2004 May; 15(5): 519-30. PubMed PMID: 15144581. *Equal contribution first authors

Lebherz C, Gao G, Louboutin JP, Millar J, Rader D, and Wilson JM. Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia. J Gene Med. 2004 Jun; 6(6): 663-72. PubMed PMID: 15170737.

Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X, and Wilson JM. Clades of adeno-associated viruses are widely disseminated in human tissues. J Virol. 2004 Jun; 78(12): 6381-8. PubMed PMID: 15163731; PMCID: PMC416542.

Reyes-Sandoval A, Fitzgerald JC, Grant R, Roy S, Xiang ZQ, Li Y, Gao GP, Wilson JM, and Ertl HC. Human immunodeficiency virus type 1-specific immune responses in primates upon sequential immunization with adenoviral vaccine carriers of human and simian serotypes. J Virol. 2004 Jul; 78(14): 7392-9. PubMed PMID: 15220412; PMCID: PMC434124.

Roy S, Gao G, Clawson DS, Vandenberghe LH, Farina SF, and Wilson JM. Complete nucleotide sequences and genome organization of four chimpanzee adenoviruses. Virology. 2004 Jul 1; 324(2): 361-72. PubMed PMID: 15207622.

Rowe T, Gao G, Hogan RJ, Crystal RG, Voss TG, Grant RL, Bell P, Kobinger GP, Wivel NA, and Wilson JM. Macaque model for severe acute respiratory syndrome. J Virol. 2004 Oct; 78(20): 11401-4. PubMed PMID: 15452262; PMCID: PMC521815.

Hogan RJ, Gao G, Rowe T, Bell P, Flieder D, Paragas J, Kobinger GP, Wivel NA, Crystal RG, Boyer J, Feldmann H, Voss TG, and Wilson JM. Resolution of primary severe acute respiratory syndrome-associated coronavirus infection requires Stat1. J Virol. 2004 Oct; 78(20): 11416-21. PubMed PMID: 15452265; PMCID: PMC521834.

Vais H, Gao GP, Yang M, Tran P, Louboutin JP, Somanathan S, Wilson JM, and Reenstra WW. Novel adenoviral vectors coding for GFP-tagged wtCFTR and deltaF508-CFTR: characterization of expression and electrophysiological properties in A549 cells. Pflugers Arch. 2004 Dec; 449(3): 278-87. PubMed PMID: 15452712.


Vinner L, Wee EG, Patel S, Corbet S, Gao GP, Nielsen C, Wilson JM, Ertl HC, Hanke T, and Fomsgaard A. Immunogenicity in Mamu-A*01 rhesus macaques of a CCR5-tropic human immunodeficiency virus type 1 envelope from the primary isolate (Bx08) after synthetic DNA prime and recombinant adenovirus 5 boost. J Gen Virol. 2003 Jan; 84(Pt 1): 203-13. PubMed PMID: 12533717.

Xiang ZQ, Gao GP, Li Y, Wilson JM, and Ertl HC. T helper cell-independent antibody responses to the transgene product of an e1-deleted adenoviral vaccine require NK11 T cells. Virology. 2003 Jan 20; 305(2): 397-405. PubMed PMID: 12573585.

Fitzgerald JC, Gao GP, Reyes-Sandoval A, Pavlakis GN, Xiang ZQ, Wlazlo AP, Giles-Davis W, Wilson JM, and Ertl HC. A simian replication-defective adenoviral recombinant vaccine to HIV-1 gag. J Immunol. 2003 Feb 1; 170(3): 1416-22. PubMed PMID: 12538702.

Johnston J, Tazelaar J, Rivera VM, Clackson T, Gao GP, and Wilson JM. Regulated expression of erythropoietin from an AAV vector safely improves the anemia of beta-thalassemia in a mouse model. Mol Ther. 2003 Apr; 7(4): 493-7. PubMed PMID: 12727112.

Varnavski AN, Schlienger K, Bergelson JM, Gao GP, and Wilson JM. Efficient transduction of human monocyte-derived dendritic cells by chimpanzee-derived adenoviral vector. Hum Gene Ther. 2003 Apr 10; 14(6): 533-44. PubMed PMID: 12718764.

Gao G, Alvira MR, Somanathan S, Lu Y, Vandenberghe LH, Rux JJ, Calcedo R, Sanmiguel J, Abbas Z, and Wilson JM. Adeno-associated viruses undergo substantial evolution in primates during natural infections. Proc Natl Acad Sci U S A. 2003 May 13; 100(10):6081-6. PubMed PMID: 12716974; PubMed Central PMCID: PMC156329.

Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP, Wilson JM, and Batshaw ML. Fatal systemic inflammatory response syndrome in an ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab. 2003 Sep-Oct; 80(1-2): 148-58. PubMed PMID: 14567964.

Gao G, Zhou X, Alvira MR, Tran P, Marsh J, Lynd K, Xiao W, and Wilson JM. High throughput creation of recombinant adenovirus vectors by direct cloning, green-white selection and I-Sce I-mediated rescue of circular adenovirus plasmids in 293 cells. Gene Ther. 2003 Oct; 10(22): 1926-30. PubMed PMID: 14502222.

Xiang ZQ, Gao GP, Reyes-Sandoval A, Li Y, Wilson JM, and Ertl HC. Oral vaccination of mice with adenoviral vectors is not impaired by preexisting immunity to the vaccine carrier. J Virol. 2003 Oct; 77(20): 10780-9. PubMed PMID: 14512528; PMCID: PMC224991.

Xiang Z, Li Y, Gao G, Wilson JM, and Ertl HC. Mucosally delivered E1-deleted adenoviral vaccine carriers induce transgene product-specific antibody responses in neonatal mice. J Immunol. 2003 Oct 15; 171(8): 4287-93. PubMed PMID: 14530353.

Pinto AR, Fitzgerald JC, Giles-Davis W, Gao GP, Wilson JM, and Ertl HC. Induction of CD8+ T cells to an HIV-1 antigen through a prime boost regimen with heterologous E1-deleted adenoviral vaccine carriers. J Immunol. 2003 Dec 15; 171(12): 6774-9. PubMed PMID: 14662882.


Raper SE, Yudkoff M, Chirmule N, Gao GP, Nunes F, Haskal ZJ, Furth EE, Propert KJ, Robinson MB, Magosin S, Simoes H, Speicher L, Hughes J, Tazelaar J, Wivel NA, Wilson JM, and Batshaw ML. A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency. Hum Gene Ther. 2002 Jan 1; 13(1): 163-75. PubMed PMID: 11779420.

Cohen CJ, Xiang ZQ, Gao GP, Ertl HC, Wilson JM, and Bergelson JM. Chimpanzee adenovirus CV-68 adapted as a gene delivery vector interacts with the coxsackievirus and adenovirus receptor. J Gen Virol. 2002 Jan; 83(Pt 1): 151-5. PubMed PMID: 11752711.

Deriy L, Chen ZC, Gao GP, and Galili U. Expression of alpha-gal epitopes on HeLa cells transduced with adenovirus containing alpha1,3galactosyltransferase cDNA. Glycobiology. 2002 Feb; 12(2): 135-44. PubMed PMID: 11886847.

Xiang Z, Gao G, Reyes-Sandoval A, Cohen CJ, Li Y, Bergelson JM, Wilson JM, and Ertl HC. Novel, chimpanzee serotype 68-based adenoviral vaccine carrier for induction of antibodies to a transgene product. J Virol. 2002 Mar; 76(6): 2667-75. PubMed PMID: 11861833; PMCID: PMC135983.

Gao GP, Lu F, Sanmiguel JC, Tran PT, Abbas Z, Lynd KS, Marsh J, Spinner NB, and Wilson JM. Rep/Cap gene amplification and high-yield production of AAV in an A549 cell line expressing Rep/Cap. Mol Ther. 2002 May; 5(5 Pt 1): 644-9. PubMed PMID: 11991756.

Varnavski AN, Zhang Y, Schnell M, Tazelaar J, Louboutin JP, Yu QC, Bagg A, Gao GP, and Wilson JM. Preexisting immunity to adenovirus in rhesus monkeys fails to prevent vector-induced toxicity. J Virol. 2002 Jun; 76(11): 5711-9. PubMed PMID: 11991999; PMCID: PMC137042.

Auricchio A, Gao GP, Yu QC, Raper S, Rivera VM, Clackson T, and Wilson JM. Constitutive and regulated expression of processed insulin following in vivo hepatic gene transfer. Gene Ther. 2002 Jul; 9(14): 963-71. PubMed PMID: 12085245.

Auricchio A, O'Connor E, Weiner D, Gao GP, Hildinger M, Wang L, Calcedo R, and Wilson JM. Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. J Clin Invest. 2002 Aug; 110(4): 499-504. PubMed PMID: 12189244; PMCID: PMC150421.

*Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, and Wilson JM. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A. 2002 Sep 3; 99(18): 11854-9. PubMed PMID: 12192090; PMCID: PMC129358.

* In two consecutive months, this paper was selected by ISI as among the most cited papers (top 1%, 10/2003) and hottest break-through papers (top 0.1%, 11/2003) in 22 broad fields of Science.

Green AP, Huang JJ, Scott MO, Kierstead TD, Beaupré I, Gao GP, and Wilson JM. A new scalable method for the purification of recombinant adenovirus vectors. Hum Gene Ther. 2002 Nov 1; 13(16): 1921-34. PubMed PMID: 12427304.


Tao N*, Gao GP*, Parr M*, Johnston J, Baradet T, Wilson JM, Barsoum J, and Fawell SE. Sequestration of adenoviral vector by Kupffer cells leads to a nonlinear dose response of transduction in liver. Mol Ther. 2001 Jan; 3(1): 28-35. PubMed PMID: 11162308. *Equal contribution first authors

Eck SL, Alavi JB, Judy K, Phillips P, Alavi A, Hackney D, Cross P, Hughes J, Gao G, Wilson JM, and Propert K. Treatment of recurrent or progressive malignant glioma with a recombinant adenovirus expressing human interferon-beta (H5010CMVhIFN-beta): a phase I trial. Hum Gene Ther. 2001 Jan 1; 12(1): 97-113. PubMed PMID: 11177547.

Auricchio A, Hildinger M, O'Connor E, Gao GP, and Wilson JM. Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column. Hum Gene Ther. 2001 Jan 1; 12(1): 71-6. PubMed PMID: 11177544.

Cordier L, Gao GP, Hack AA, McNally EM, Wilson JM, Chirmule N, and Sweeney HL. Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies. Hum Gene Ther. 2001 Jan 20; 12(2): 205-15. PubMed PMID: 11177557.

Dejneka NS, Auricchio A, Maguire AM, Ye X, Gao GP, Wilson JM, and Bennett J. Pharmacologically regulated gene expression in the retina following transduction with viral vectors. Gene Ther. 2001 Mar; 8(6): 442-6. PubMed PMID: 11313822.

Zhang Y, Chirmule N, Gao GP, Qian R, Croyle M, Joshi B, Tazelaar J, and Wilson JM. Acute cytokine response to systemic adenoviral vectors in mice is mediated by dendritic cells and macrophages. Mol Ther. 2001 May; 3(5 Pt 1): 697-707. PubMed PMID: 11356075.

Schnell MA, Zhang Y, Tazelaar J, Gao GP, Yu QC, Qian R, Chen SJ, Varnavski AN, LeClair C, Raper SE, and Wilson JM. Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors. Mol Ther. 2001 May; 3(5 Pt 1): 708-22. PubMed PMID: 11356076.

Zoltick PW, Chirmule N, Schnell MA, Gao GP, Hughes JV, and Wilson JM. Biology of E1-deleted adenovirus vectors in nonhuman primate muscle. J Virol. 2001 Jun; 75(11): 5222-9. PubMed PMID: 11333904; PMCID: PMC114928.

Hildinger M, Auricchio A, Gao G, Wang L, Chirmule N, and Wilson JM. Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer. J Virol. 2001 Jul; 75(13): 6199-203. PubMed PMID: 11390622; PMCID: PMC114336.

Odaka M, Sterman DH, Wiewrodt R, Zhang Y, Kiefer M, Amin KM, Gao GP, Wilson JM, Barsoum J, Kaiser LR, and Albelda SM. Eradication of intraperitoneal and distant tumor by adenovirus-mediated interferon-beta gene therapy is attributable to induction of systemic immunity. Cancer Res. 2001 Aug 15; 61(16): 6201-12. PubMed PMID: 11507073.

Farina SF*, Gao GP*, Xiang ZQ, Rux JJ, Burnett RM, Alvira MR, Marsh J, Ertl HC, and Wilson JM. Replication-defective vector based on a chimpanzee adenovirus. J Virol. 2001 Dec; 75(23): 11603-13. PubMed PMID: 11689642; PMCID: PMC114747. *Equal contribution first authors


Gao GP, Engdahl RK, and Wilson JM. A cell line for high-yield production of E1-deleted adenovirus vectors without the emergence of replication-competent virus. Hum Gene Ther. 2000 Jan 1; 11(1): 213-9. PubMed PMID: 10646652.

Cordier L, Hack AA, Scott MO, Barton-Davis ER, Gao G, Wilson JM, McNally EM, and Sweeney HL. Rescue of skeletal muscles of gamma-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer. Mol Ther. 2000 Feb; 1(2): 119-29. PubMed PMID: 10933922.

Sarkar R, Gao GP, Chirmule N, Tazelaar J, and Kazazian HH Jr. Partial correction of murine hemophilia A with neo-antigenic murine factor VIII. Hum Gene Ther. 2000 Apr 10; 11(6): 881-94. PubMed PMID: 10779165.

Watanabe S, Imagawa T, Boivin GP, Gao G, Wilson JM, and Hirsch R. Adeno-associated virus mediates long-term gene transfer and delivery of chondroprotective IL-4 to murine synovium. Mol Ther. 2000 Aug; 2(2): 147-52. PubMed PMID: 10947942.

Zhang Y, Chirmule N, Gao G, and Wilson J. CD40 ligand-dependent activation of cytotoxic T lymphocytes by adeno-associated virus vectors in vivo: role of immature dendritic cells. J Virol. 2000 Sep; 74(17): 8003-10. PubMed PMID: 10933709; PMCID: PMC112332.

Chen SJ, Rader DJ, Tazelaar J, Kawashiri M, Gao G, and Wilson JM. Prolonged correction of hyperlipidemia in mice with familial hypercholesterolemia using an adeno-associated viral vector expressing very-low-density lipoprotein receptor. Mol Ther. 2000 Sep; 2(3): 256-61. PubMed PMID: 10985956.

Gao G, Qu G, Burnham MS, Huang J, Chirmule N, Joshi B, Yu QC, Marsh JA, Conceicao CM, and Wilson JM. Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo. Hum Gene Ther. 2000 Oct 10; 11(15): 2079-91. PubMed PMID: 11044910.


Ye X, Rivera VM, Zoltick P, Cerasoli F Jr, Schnell MA, Gao G, Hughes JV, Gilman M, and Wilson JM. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science. 1999 Jan 1; 283(5398): 88-91. PubMed PMID: 9872748.

Lanuti M, Gao GP, Force SD, Chang MY, El Kouri C, Amin KM, Hughes JV, Wilson JM, Kaiser LR, and Albelda SM. Evaluation of an E1E4-deleted adenovirus expressing the herpes simplex thymidine kinase suicide gene in cancer gene therapy. Hum Gene Ther. 1999 Feb 10; 10(3): 463-75. PubMed PMID: 10048398.

Greelish JP, Su LT, Lankford EB, Burkman JM, Chen H, Konig SK, Mercier IM, Desjardins PR, Mitchell MA, Zheng XG, Leferovich J, Gao GP, Balice-Gordon RJ, Wilson JM, and Stedman HH. Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector. Nat Med. 1999 Apr; 5(4): 439-43. PubMed PMID: 10202936.

Xiao W, Chirmule N, Berta SC, McCullough B, Gao G, and Wilson JM. Gene therapy vectors based on adeno-associated virus type 1. J Virol. 1999 May; 73(5): 3994-4003. PubMed PMID: 10196295; PMCID: PMC104178.

Chirmule N, Truneh A, Haecker SE, Tazelaar J, Gao G, Raper SE, Hughes JV, and Wilson JM. Repeated administration of adenoviral vectors in lungs of human CD4 transgenic mice treated with a nondepleting CD4 antibody. J Immunol. 1999 Jul 1; 163(1): 448-55. PubMed PMID: 10384148.

Bennett J, Maguire AM, Cideciyan AV, Schnell M, Glover E, Anand V, Aleman TS, Chirmule N, Gupta AR, Huang Y, Gao GP, Nyberg WC, Tazelaar J, Hughes J, Wilson JM, and Jacobson SG. Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natl Acad Sci U S A. 1999 Aug 17; 96(17): 9920-5. PubMed PMID: 10449795; PMCID: PMC22311.

Grifman M, Chen NN, Gao GP, Cathomen T, Wilson JM, and Weitzman MD. Overexpression of cyclin A inhibits augmentation of recombinant adeno-associated virus transduction by the adenovirus E4orf6 protein. J Virol. 1999 Dec; 73(12): 10010-9. PubMed PMID: 10559315; PubMed Central PMCID: PMC113052.


Raper SE, Haskal ZJ, Ye X, Pugh C, Furth EE, Gao GP, and Wilson JM. Selective gene transfer into the liver of non-human primates with E1-deleted, E2A-defective, or E1-E4 deleted recombinant adenoviruses. Hum Gene Ther. 1998 Mar 20; 9(5): 671-9. PubMed PMID: 9551615.

Chirmule N, Hughes JV, Gao GP, Raper SE, and Wilson JM. Role of E4 in eliciting CD4 T-cell and B-cell responses to adenovirus vectors delivered to murine and nonhuman primate lungs. J Virol. 1998 Jul; 72(7): 6138-45. PubMed PMID: 9621078; PMCID: PMC110420.

Ye X, Gao GP, Pabin C, Raper SE, and Wilson JM. Evaluating the potential of germ line transmission after intravenous administration of recombinant adenovirus in the C3H mouse. Hum Gene Ther. 1998 Sep 20; 9(14): 2135-42. PubMed PMID: 9759939.

Gao GP, Qu G, Faust LZ, Engdahl RK, Xiao W, Hughes JV, Zoltick PW, and Wilson JM. High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus. Hum Gene Ther. 1998 Nov 1; 9(16): 2353-62. PubMed PMID: 9829534.


Fisher KJ, Gao GP, Weitzman MD, DeMatteo R, Burda JF, and Wilson JM. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol. 1996 Jan; 70(1): 520-32. PubMed PMID: 8523565; PMCID: PMC189840.

Gao GP and Herrera RJ. Enrichment of middle repetitive element Bm-1 transcripts in translationally active RNA fractions of the silkmoth, Bombyx mori. Genetica. 1996 Mar; 97(2): 173-82. PubMed PMID: 8901137.

Kaul R, Gao GP, Matalon R, Aloya M, Su Q, Jin M, Johnson AB, Schutgens RB, and Clarke JT. Identification and expression of eight novel mutations among non-Jewish patients with Canavan disease. Am J Hum Genet. 1996 Jul; 59(1): 95-102. PubMed PMID: 8659549; PMCID: PMC1915091. *GGP’s PhD thesis advisor, publications generated from GGP thesis research.

Gao GP, Yang Y, and Wilson JM. Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy. J Virol. 1996 Dec; 70(12): 8934-43. PubMed PMID: 8971023; PMCID: PMC190991.


Matalon R, Kaul R*, Gao GP, Michals K, Gray RG, Bennett-Briton S, Norman A, Smith M, and Jakobs C. Prenatal diagnosis for Canavan disease: the use of DNA markers. J Inherit Metab Dis. 1995; 18(2): 215-7. PubMed PMID: 7564250.

Kaul R*, Gao GP, Michals K, Whelan DT, Levin S, and Matalon R. Novel (cys152 > arg) missense mutation in an Arab patient with Canavan disease. Hum Mutat. 1995; 5(3): 269-71. PubMed PMID: 7599639. *GGP’s PhD thesis advisor, publications generated from GGP thesis research.


Kaul R*, Gao GP, Balamurugan K, and Matalon R. Canavan disease: molecular basis of aspartoacylase deficiency. J Inherit Metab Dis. 1994; 17(3): 295-7. PubMed PMID: 7528829. *GGP’s PhD thesis advisor, publications generated from GGP thesis research.

Kaul R*, Balamurugan K, Gao GP, and Matalon R. Canavan disease: genomic organization and localization of human ASPA to 17p13-ter and conservation of the ASPA gene during evolution. Genomics. 1994 May 15; 21(2): 364-70. PubMed PMID: 8088831.

Kaul R*, Gao GP, Aloya M, Balamurugan K, Petrosky A, Michals K, and Matalon R. Canavan disease: mutations among Jewish and non-Jewish patients. Am J Hum Genet. 1994 Jul; 55(1): 34-41. PubMed PMID: 8023850; PMCID: PMC1918221. *GGP’s PhD thesis advisor, publications generated from GGP thesis research.


Kaul R*, Gao GP, Balamurugan K, and Matalon R. Cloning of the human aspartoacylase cDNA and a common missense mutation in Canavan disease. Nat Genet. 1993 Oct; 5 (2): 118-23. PubMed PMID: 8252036.  *GGP’s PhD thesis advisor, publications generated from GGP thesis research


von Sternberg RM, Novick GE, Gao GP, and Herrera RJ. Genome canalization: the coevolution of transposable and interspersed repetitive elements with single copy DNA. Genetica. 1992; 86 (1-3): 215-46. PubMed PMID: 1334910. 

Books & Chapters

Wivel N, Gao GP, and Wilson JM.  “Adenoviral Vectors” in The Development of Human Gene Therapy.  Edited by Theodore Friedman, Cold Spring Harbor Laboratory Press, New York, pp 87-110, 1999.


Gao GP, Wivel N, and Wilson JM.  “Production of Recombinant AAV, Viral Vectors for Gene Therapy” in Advances in Virus Research.  Edited by JC Glorioso, Academic Press, New York, 55:529-543, 2000.

Gao GP, Vandenberghe LH, and Wilson JM.  “Identification of Novel AAV Serotypes for Use as Vectors” in Gene Therapy in the Brain, Edited by Michael G. Kaplitt and Matthew During, 2006.

Zhi Y and Gao G. “Muscle as a Target for Genetic Vaccine” in Muscle Gene Therapy.  Edited by D Duan, Springer Science + Business Media, LLC, pp 231-247, 2010.

Gao GP, Mueller C and Flotte TR.  “AAV as a gene therapy vector” in Regenerative Therapy for the Musculoskeletal System Using Recombinant Adeno-Associated Viral Vectors.  Edited by Magali Cucchiarini and Henning Madry, Research Signpost, Kerala, India, pp. 1-22, 2010.

Gao GP, and Sena-Esteves M.  “Introducing Genes into Mammalian Cells: Viral Vectors” in Molecular Cloning, Vol 2: A Laboratory Manual.  Edited by MR Green and J Sambrook, Cold Spring Harbor Laboratory Press, New York, pp 1209-1313, 2012.


Dr. Guangping Gao from the Gao Lab (Horae Gene Therapy Center) is conducting research and developing therapeutic strategies for rare inherited diseases such as the Canavan disease.

Guangping Gao, PhD


Visit Lab Website Contact Dr. Gao

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