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Publications

You can check out our papers on NCBI or in the list below.

2024

Liu H, Zhang Y, Yip M, Ren L, Liang J, Chen X, Liu N, Du A, Wang J, Chang H, Oh H, Zhou C, Xing R, Xu M, Guo P, Gessler D, Xie J, Tai PWL, Gao G, Wang D. Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection. Mol Ther Methods Clin Dev. 2024

2023

Chang H, Du A, Jiang J, Ren L, Liu N, Zhou X, Liang J, Gao G, Wang D. Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice. Mol Ther Methods Clin Dev. 2023

Xu L, Wang D, Zhao L, Yang Z, Liu X, Li X, Yuan T, Wang Y, Huang T, Bian N, He Y, Chen X, Tian B, Liu Z, Luo F, Si W, Gao G, Ji W, Niu Y, Wei J. C9orf72 poly(PR) aggregation in nucleus induces ALS/FTD-related neurodegeneration in cynomolgus monkeys. Neurobiol Dis. 2023

Vansandt LM, Meinsohn MC, Godin P, Nagykery N, Sicher N, Kano M, Kashiwagi A, Chauvin M, Saatcioglu HD, Barnes JL, Miller AG, Thompson AK, Bateman HL, Donelan EM, González R, Newsom J, Gao G, Donahoe PK, Wang D, Swanson WF, Pépin D. Durable contraception in the female domestic cat using viral-vectored delivery of a feline anti-Müllerian hormone transgene. Nat Commun. 2023

Yang H, Brown R, Wang D, Strauss KA, Gao G. Rescue of GM3 synthase deficiency by spatially controlled rAAV-mediated ST3GAL5 delivery. JCI Insight. 2023

Arbab M, Matuszek Z, Kray KM, Du A, Newby GA, Blatnik AJ, Raguram A, Richter MF, Zhao KT, Levy JM, Shen MW, Arnold WD, Wang D, Xie J, Gao G, Burghes AHM, Liu DR. Base editing rescue of spinal muscular atrophy in cells and in mice. Science. 2023

2022

Wang D, Xue X, Gunn G, Du M, Siddiqui A, Weetall M, Keeling KM. Ataluren suppresses a premature termination codon in an MPS I-H mouse. J Mol Med (Berl). 2022

Davidson BL, Gao G, Berry-Kravis E, Bradbury A, Bönnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz A, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CJ, Sahin M. Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders. Mol Ther. 2022

Wang J, Zhang Y, Mendonca CA, Yukselen O, Muneeruddin K, Ren L, Liang J, Zhou C, Xie J, Li J, Jiang Z, Kucukural A, Shaffer SA, Gao G, Wang D. AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice. Nature. 2022 (UMass Chan News, Research Highlight in Nature Reviews Drug Discovery, News and Views by Dr. Barrie Carter in Human Gene Therapy).

2021

Ibraheim R, Tai PWL, Mir A, Javeed N, Wang J, Rodríguez TC, Namkung S, Nelson S, Khokhar ES, Mintzer E, Maitland S, Chen Z, Cao Y, Tsagkaraki E, Wolfe SA, Wang D, Pai AA, Xue W, Gao G, Sontheimer EJ. Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo. Nat Commun. 2021 (biorxiv version)

Liang SQ, Walkey CJ, Martinez AE, Su Q, Dickinson ME, Wang D, Lagor WR, Heaney JD, Gao G, Xue W. AAV5 delivery of CRISPR/Cas9 supports effective genome editing in mouse lung airway. Mol Ther. 2021

Wang D. Juggling Safety and Efficacy: Finding Ways to Achieve Both. Hum Gene Ther. 2021

Meinsohn MC, Saatcioglu HD, Wei L, Li Y, Horn H, Chauvin M, Kano M, Nguyen NMP, Nagykery N, Kashiwagi A, Samore WR, Wang D, Oliva E, Gao G, Morris ME, Donahoe PK, Pépin D. Single-cell sequencing reveals suppressive transcriptional programs regulated by MIS/AMH in neonatal ovaries. Proc Natl Acad Sci U S A. 2021

Yang H, Brown RH Jr, Wang D, Strauss KA, Gao G. AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies. Trends Mol Med. 2021

2020

Tran NT, Heiner C, Weber K, Weiand M, Wilmot D, Xie J, Wang D, Brown A, Manokaran S, Su Q, Zapp ML, Gao G, Tai PWL. AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity. Mol Ther Methods Clin Dev. 2020

Yang YS, Xie J, Chaugule S, Wang D, Kim JM, Kim J, Tai PWL, Seo SK, Gravallese E, Gao G, Shim JH. Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy. Mol Ther Methods Clin Dev. 2020

Wang D, Zhang F, Gao G. CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors. Cell. 2020

2019

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

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

Saatcioglu HD, Kano M, Horn H, Zhang L, Samore W, Nagykery N, Meinsohn MC, Hyun M, Suliman R, Poulo J, Hsu J, Sacha C, Wang D, Gao G, Lage K, Oliva E, Morris Sabatini ME, Donahoe PK, Pépin D. Single-cell sequencing of neonatal uterus reveals an Misr2+ endometrial progenitor indispensable for fertility. Elife. 2019

Xiao Y*, Muhuri M*, Li S*, Qin W, Xu G, Luo L, Li J, Letizia AJ, Wang SK, Chan YK, Wang C, Fuchs SP, Wang D, Su Q, Nahid MA, Church GM, Farzan M, Yang L, Wei Y, Desrosiers RC, Mueller C, Tai PW, Gao G. Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity. JCI Insight. 2019

Gong Y, Berenson A, Laheji F, Gao G, Wang D, Ng C, Volak A, Kok R, Kreouzis V, Dijkstra IM, Kemp S, Maguire CA, Eichler F. Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy. Hum Gene Ther. 2019

Wang D*, Tai PWL*, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019

2018

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. Mol Ther Methods Clin Dev. 2018

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

Song CQ, Wang D, Jiang T, O'Connor K, Tang Q, Cai L, Li X, Weng Z, Yin H, Gao G, Mueller C, Flotte TR, Xue W. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency. Hum Gene Ther. 2018

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-Detargeted Gene Delivery in Neonates. Mol Ther Methods Clin Dev. 2018

Wang D*, Li J*, Tran K*, Burt DR, Zhong L, Gao G. Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space. Hum Gene Ther Methods. 2018

Wang D, Gao G. Taking a Hint from Structural Biology: To Better Understand AAV Transport across the BBB. Mol Ther. 2018

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

2017

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. Neuroscience. 2017

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

Kano M, Sosulski AE, Zhang L, Saatcioglu HD, Wang D, Nagykery N, Sabatini ME, Gao G, Donahoe PK, Pépin D. AMH/MIS as a contraceptive that protects the ovarian reserve during chemotherapy. Proc Natl Acad Sci U S A. 2017

2016

Murlidharan G, Sakamoto K, Rao L, Corriher T, Wang D, Gao G, Sullivan P, Asokan A. CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector. Mol Ther Nucleic Acids. 2016

Guo Y, Wang D, Qiao T, Yang C, 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

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

2015

Pépin D, Sosulski A, Zhang L, Wang D, Vathipadiekal V, Hendren K, Coletti CM, Yu A, Castro CM, Birrer MJ, Gao G, 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

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

2014

Wang D, Gao G. State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications. Discov Med. 2014

Wang D, Gao G. State-of-the-art human gene therapy: part I. Gene delivery technologies. Discov Med. 2014

Wang D*, Zhong L*, Nahid MA*, Gao G. The potential of adeno-associated viral vectors for gene delivery to muscle tissue. Expert Opin Drug Deliv. 2014

2010-2013

Keeling KM, Wang D, Dai Y, Murugesan S, Chenna B, Clark J, Belakhov V, Kandasamy J, Velu SE, Baasov T, Bedwell DM. Attenuation of nonsense-mediated mRNA decay enhances in vivo nonsense suppression. PLoS One. 2013

Keeling KM, Wang D, Conard SE, Bedwell DM. Suppression of premature termination codons as a therapeutic approach. Crit Rev Biochem Mol Biol. 2012

Wang D, Belakhov V, Kandasamy J, Baasov T, Li SC, Li YT, Bedwell DM, Keeling KM. The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse. Mol Genet Metab. 2012

Wang D, Shukla C, Liu X, Schoeb TR, Clarke LA, Bedwell DM, Keeling KM. Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation. Mol Genet Metab. 2010