Publications
You can check out our papers on NCBI or in the list below.
2024
Du A, Yang K, Zhou X, Ren L, Liu N, Zhou C, Liang J, Yan N, Gao G, Wang D. Systemic gene therapy corrects neurological phenotype in a mouse model of NGLY1 deficiency. JCI Insight. 2024
Wang J, Gao G, Wang D. Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders. Neurotherapeutics. 2024
Liang SQ, Navia AW, Ramseier M, Zhou X, Martinez M, Lee C, Zhou C, Wu J, Xie J, Su Q, Wang D, Flotte TR, Anderson DG, Tarantal AF, Shalek AK, Gao G, Xue W. AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys. Hum Gene Ther. 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
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*, 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
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
2016
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
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
