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  • Werneburg S, Jung J, Kunjamma RB, Ha SK, Luciano NJ, Willis CM, Gao G, Biscola NP, Havton LA, Crocker SJ, Popko B, Reich DS, Schafer DP. Targeted Complement Inhibition at Synapses Prevents Microglial Synaptic Engulfment and Synapse Loss in Demyelinating Disease. Immunity. 2019 Dec 23. PMID: 31883839.
  • Xie J, Tai PWL, Brown A, Gong S, Zhu S, Wang Y, Li C, Colpan C, Su Q, He R, Ma H, Li J, Ye H, Ko J, Zamore PD, Gao G. Effective and Accurate Gene Silencing by a Recombinant AAV-Compatible MicroRNA Scaffold. Mol Ther. 2019 Nov 27. PMID: 31843447.
  • Wang D, Niu Y, Ren L, Kang Y, Tai PWL, Si C, Mendonca CA, Ma H, Gao G, Ji W. Gene Delivery to Nonhuman Primate Preimplantation Embryos Using Recombinant Adeno-Associated Virus. Adv Sci (Weinh). 2019 Nov 06; 6(21):1900440. PMID: 31728271.
  • Sweeney CG, Kearney PJ, Fagan RR, Smith LA, Bolden NC, Zhao-Shea R, Rivera IV, Kolpakova J, Xie J, Gao G, Tapper AR, Martin GE, Melikian HE. Conditional, inducible gene silencing in dopamine neurons reveals a sex-specific role for Rit2 GTPase in acute cocaine response and striatal function. Neuropsychopharmacology. 2019 Jul 05. PMID: 31277075.
  • Yang YS, Xie J, Wang D, Kim JM, Tai PWL, Gravallese E, Gao G, Shim JH. Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. Nat Commun. 2019 Jul 04; 10(1):2958. PMID: 31273195.
  • Xu M, Li J, Xie J, He R, Su Q, Gao G, Tai PWL. High-throughput quantification of in vivo AAV transduction with barcoded non-coding RNAs. Hum Gene Ther. 2019 May 09. PMID: 31072208.
  • Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019 Feb 01. PMID: 30710128.
  • Gessler DJ, Tai PWL, Li J, Gao G. Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System. Methods Mol Biol. 2019; 1950:143-163. PMID: 30783972.
  • Newman H, Hu J, Li X, He J, Bradford L, Shan S, Wu X, Zhu B, Yang W, Fu B, Zhu B, Gao G. Evaluation of portable colposcopy and human papillomavirus testing for screening of cervical cancer in rural China. Int J Gynecol Cancer. 2019 Jan; 29(1):23-27. PMID: 30640679.


  • Luo X, Zhang D, Xie J, Su Q, He X, Bai R, Gao G*, Pan W*. MicroRNA-96 Promotes Schistosomiasis Hepatic Fibrosis in mice by suppressing Smad7. Molec Ther Methods Clin Dev. 2018 Oct 10; 11:73-82. PubMed PMID: 30406154. PMCID: PMC6214875. *Co-corresponding Authors.
  • Wang D, Zhong L, Li M, Li J, Tran K, Ren L, He R, Xie J, Moser RP, Fraser C, Kuchel T, Sena-Esteves M, Flotte TR, Aronin N, Gao G. Adeno-associated virus neutralizing antibodies in large animals and their impact on brain intraparenchymal gene transfer. Molec Ther Methods Clin Dev 2018 Oct 4; 11:65-72. PubMed PMID: 30397628. PMCID: PMC6205343.
  • Wang D, Li J, Song CQ, Tran K, Mou H, Wu PH, Tai PWL, Mendonca CA, Ren L, Wang BY, Su Q, Gessler DJ, Zamore PD*, Xue W*, Gao G*. Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice. Nat Biotechnol 2018 Oct; 36(9): 839-842. PubMed PMID: 30102296. PMCID: PMC6126964. *Co-corresponding authors.
  • Sena-Esteves M, Gao G. Monitoring lentivirus vector stocks for replication-competent viruses. Cold Spring Harb Protoc. 2018 Apr 2; 2018(4): pdb.prot095703. PubMed PMID: 29610364.
  • Sena-Esteves M, Gao G. Titration of lentivirus vectors. Cold Spring Harb Protoc. 2018 Apr 2; 2018 (4): pdb.prot095695. PubMed PMID: 29610363.
  • Sena-Esteves M, Gao G. Production of high-titer retrovirus and lentivirus vectors. Cold Spring Harb Protoc. 2018 Apr 2; 2018 (4): pdb.prot095687. PubMed PMID: 29610362.
  • Wang D, Li J, Tran K, Burt DR, Gao G. Slow infusion of recombinant adeno-associated viruses into the mouse cerebrospinal fluid space. Hum Gene Ther Methods. 2018 Apr; 29(2): 75-85. PubMed PMID: 295996011. PMCID: PMC5910051.
  • Wang D, Li S, Gessler DJ, Xie J, Zhong L, Li J, Tran K, Van Vliet K, Ren L, Su Q, He R, Goetzmann JE, Flotte TR, Agbandje-McKenna M, Gao G. A rationally engineered capsid variant of AAV9 for systemic CNS-directed and peripheral tissue-targeted gene delivery in neonates. Mol Ther Methods Clin Dev. 2018 Mar 16; 9:234-246. PubMed PMID: 29766031. PMCID: PMC5948233.
  • He X, Xie J, Wang Y, Fan X, Su Q, Sun Y, Lei N, Zhang D, Gao G*, Pan W*. Down-regulation of microRNA-203-3p initiates type 2 pathology during schistosome infection via elevation of interleukin-33. PLoS Pathog. 2018 Mar 19; 14(3):e1006957. PubMed PMID: 29554131. PMCID: PMC5875897. *Co-corresponding authors.
  • Lu Y, Tai PWL, Ai J, Gessler DJ, Su Q, Yao X, Zheng Q, Zamore PD, Xu X, Gao G. Transcriptome profiling of neovascularized corneas reveals miR-204 as a multi-target biotherapy deliverable by rAAVs. Mol Ther Nucleic Acids. 2018 Mar 2; 10:349-360. PubMed PMID: 29499946. PMCID: PMC5862543.
  • Flotte TR, Gao G. Gene and cell therapy in China: highlighting excellence in the 21st century : 21. Hum Gene Ther 2018 Feb; 29(2): 97. PubMed PMID: 29446999. PMCID: N/A (Editorial).
  • Wang D, Gao G. Taking a hint from structural biology: to better understand AAV transport across the BBB. Mol Ther. 2018 Feb 7; 26(2): 336-338. PubMed PMID: 29398483. PMCID: PMC5835224.
  • Tai PWL, Xie J, Fong K, Seetin M, Heiner C, Su Q, Weiand M, Wilmot D, Zapp M, Gao G. Adeno-associated virus genome population sequencing achieves full vector genome resolution and reveals human-vector chimeras. Mol Ther Methods Clin Dev. 2018 Feb 13; 9:130-141. PubMed PMID: 29766023. PMCID: PMC5948225.
  • Yoon Y, Wang D, Tai, PWL, Riley J, Gao G*, Rivera-Pérez JA*. Streamlined ex vivo and in vivo genome editing in mouse embryos using recombinant adeno-associated viruses. Nat Commun. 2018 Jan 29; 9(1): 412. PubMed PMID: 29379011. PMCID: PMC5788975. *Co-corresponding authors.


  • Li D, Liu C, Yang C, Wang D, Wu D, Qi Y, Su Q, Gao G, Xu Z, Guo Y. Slow intrathecal injection of rAAVrh10 enhances its transduction of spinal cord and therapeutic efficacy in a mutant SOD1 model of ALS. Neurosci. 2017 Dec 4; 365: 192-205. PubMed PMID: 29024785. PMCID: PMC5757625.
  • Ai J, Tai PWL, Lu Y, Li J, Ma H, Su Q, Wei W, Li H, and Gao G. Characterization of adenoviral transduction profile in prostate cancer cells and normal prostate tissue. Prostate. 2017 Sep; 77(12): 1265-1270. PubMed PMID: 28726259. PMCID: PMC5576497.
  • Ai J, Ibraheim R, Tai PWL, Gao G. A scalable and accurate method for quantifying vector genomes of recombinant adeno-associated viruses in crude lysate. Hum Gene Ther Methods. 2017 Jun; 28(3): 139-147. PubMed PMID: 28488944. PMCID: PMC5488319.
  • Xie J, Mao Q, Tai PWL, He R, Ai J, Su Q, Zhu Y, Ma H, Li J, Gong S, Wang D, Gao Z, Li M, Zhong L, Zhou H, Gao G. Short DNA hairpins compromise recombinant adeno-associated virus genome homogeneity. Mol Ther. 2017 Jun 7; 25(6): 1363-1374. PubMed PMID: 28462820. PMCID: PMC5474962.
  • Flotte TR, Gao G. AAV is now a medicine: we had better get this right. Hum Gene Ther. 2017 Apr; 28(4): 307. PubMed PMID: 28414552. PMCID: N/A (Editorial).
  • Gessler DJ, Li D, Xu H, Su Q, Sanmiguel J, Tuncer S, Moore C, King J, Matalon R, Gao G. Redirecting N-acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease. JCI Insight 2017 Feb 9;2(3):e90807. PubMed PMID: 28194442. PMCID: PMC5291725.
  • Ai J, Li J, Gessler D, Su Q, Wei Q, Li H, Gao G. Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery. Sci Rep 2017 Jan 9;7:40336. doi: 10.1038/srep40336. PubMed PMID: 28067312. PMCID: PMC5220346.


  • 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. Hum Gene Ther. 2016 Aug; 27(8): 598-608. PubMed PMID: 27001051. PMCID: N/A.
  • 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. PubMed PMID: 26050084. PMCID: PMC4671832. *Co-corresponding authors.
  • 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.
  • 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: PMC4961359.
  • 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.
  • 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: N/A.

Seminal Reports

  • 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, Gao G. Long-term, efficient inhibition of microRNA function in mice using rAAV vectors. Nat Methods. 2012 Mar 4;9(4):403-9. doi: 10.1038/nmeth.1903. PMID: 22388288. PMCID: PMC3420816
  • Gao G, Lu Y, Calcedo R, Grant RL, Bell P, Wang L, Figueredo J, Lock M, Wilson JM. Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. Mol Ther. 2006 Jan;13(1):77-87. Epub 2005 Oct 10. PMID: 16219492
  • Gao G, Lebherz C, Weiner DJ, Grant R, Calcedo R, McCullough B, Bagg A, Zhang Y, Wilson JM. Erythropoietin gene therapy leads to autoimmune anemia in macaques. Blood. 2004 May 1;103(9):3300-2. Epub 2003 Dec 24. PMID: 14695227
  • Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X, Wilson JM. Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol. 2004 Jun;78(12):6381-8. PMID: 15163731. PMCID: PMC416542
  • Gao G, Alvira MR, Somanathan S, Lu Y, Vandenberghe LH, Rux JJ, Calcedo R, Sanmiguel J, Abbas Z, 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. Epub 2003 Apr 25. PMID: 12716974. PMCID: PMC156329
  • Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, 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. Epub 2002 Aug 21. PMID: 12192090. PMCID: PMC129358
  • Gao GP, Qu G, Faust LZ, Engdahl RK, Xiao W, Hughes JV, Zoltick PW, 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. PMID: 9829534
  • Gao GP, Yang Y, Wilson JM. Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy. J Virol. 1996 Dec;70(12):8934-43. PMID: 8971023. PMCID: PMC190991
  • Kaul R, Gao GP, Aloya M, Balamurugan K, Petrosky A, Michals K, Matalon R. Canavan disease: mutations among Jewish and non-Jewish patients. Am J Hum Genet. 1994 Jul;55(1):34-41. PMID: 8023850. PMCID: PMC1918221
  • Kaul R, Gao GP, Balamurugan K, Matalon R. Cloning of the human aspartoacylase cDNA and a common missense mutation in Canavan disease. Nat Genet. 1993 Oct;5(2):118-23. PMID: 8252036