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Guangping Gao PhD

TitleProfessor
Endowed TitlePenelope Booth Rockwell Chair in Biomedical Research 
InstitutionUMass Chan Medical School
DepartmentMicrobiology and Physiological Systems
AddressUMass Chan Medical School
368 Plantation Street AS6-2049
Worcester MA 01605
Phone508-856-3563
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    Other Positions
    InstitutionT.H. Chan School of Medicine
    DepartmentGene Therapy Center

    InstitutionT.H. Chan School of Medicine
    DepartmentMicrobiology and Physiological Systems

    InstitutionT.H. Chan School of Medicine
    DepartmentNeuroNexus Institute

    InstitutionT.H. Chan School of Medicine
    DepartmentRNA Therapeutics Institute

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentImmunology and Microbiology Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentInterdisciplinary Graduate Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentMD/PhD Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentMillennium MD/PhD Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentNeuroscience

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentPostbaccalaureate Research Education Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentTranslational Science

    InstitutionUMass Chan Programs, Centers and Institutes
    DepartmentGene Therapy Center


    Collapse Biography 
    Collapse education and training
    West China University of Medicine, Chengdu, , ChinaMBBSMedicine & Chemisty
    Florida International University, Miami, FL, United StatesMSBiology
    Florida International University, Miami, FL, United StatesPHDBiological Sciences

    Collapse Overview 
    Collapse Summary
    Focus: Adeno-associated virus vector mediated gene transfer for gene therapy, gene therapy of inherited neurodegenerative Canavan Disease
    Collapse overview

    Academic Background

    B.A. West China Univ. of Med. Sciences, Chengdu, China
    M.S. Florida International University, Miami, FL
    Ph.D. Florida International University, Miami, FL
    Postdoctoral training at the University of Pennsylvania - School of Medicine

    Overview:

    Research in the Gao Lab primarily involves the discovery, development and use of adeno-associated virus vectors for gene therapy of genetic diseases and the study of miRNA functions in mammals. The lab works on isolation, characterization and vectorology of novel AAV vectors from primate tissues, molecular mechanisms of AAV evolution and diversity, and molecular interactions between endogenous AAV, AAV vector, host genomes and innate RNAi defense pathways. We also develop novel strategies for rAAV gene therapy of an inherited neurodegenerative disease, Canavan Disease, using novel AAVs that can cross the blood-brain-barrier for efficient CNS gene delivery and endogenous miRNA-mediated posttranscriptional de-targeting. Another area of research interest in the lab is to explore AAV vectors for delivery of denovo synthesized biological miRNA antagonists or over expression of microRNAs to elucidate micro RNA functions in adult mammals.



    Collapse Bibliographic 
    Collapse selected publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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    PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. 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 Dec 14; 31:101129. PMID: 37886602.
      Citations:    
    2. Yang YS, Lin C, Ma H, Xie J, Kaplan FS, Gao G, Shim JH. AAV-Mediated Targeting of the Activin A-ACVR1R206H Signaling in Fibrodysplasia Ossificans Progressiva. Biomolecules. 2023 09 08; 13(9). PMID: 37759764.
      Citations:    Fields:    Translation:HumansAnimalsCells
    3. Yip M, Chen J, Zhi Y, Tran NT, Namkung S, Pastor E, Gao G, Tai PWL. Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing. Viruses. 2023 05 24; 15(6). PMID: 37376529.
      Citations:    Fields:    Translation:Cells
    4. Stavre Z, Kim JM, Yang YS, N?ndel K, Chaugule S, Sato T, Park KH, Gao G, Gravallese EM, Shim JH. Schnurri-3 inhibition suppresses bone and joint damage in models of rheumatoid arthritis. Proc Natl Acad Sci U S A. 2023 05 09; 120(19):e2218019120. PMID: 37141171.
      Citations: 1     Fields:    Translation:HumansAnimalsCells
    5. Lotun A, Li D, Xu H, Su Q, Tuncer S, Sanmiguel J, Mooney M, Baer CE, Ulbrich R, Eyles SJ, Strittmatter L, Hayward LJ, Gessler DJ, Gao G. Renewal of oligodendrocyte lineage reverses dysmyelination and CNS neurodegeneration through corrected N-acetylaspartate metabolism. Prog Neurobiol. 2023 Jul; 226:102460. PMID: 37149081.
      Citations:    Fields:    Translation:AnimalsCells
    6. Bamidele N, Zhang H, Dong X, Gaston N, Cheng H, Kelly K, Watts JK, Xie J, Gao G, Sontheimer EJ. Engineering Nme2Cas9 Adenine Base Editors with Improved Activity and Targeting Scope. bioRxiv. 2023 Apr 18. PMID: 37131611.
      Citations:    
    7. Zhang H, Kelly K, Lee J, Echeverria D, Cooper D, Panwala R, Chen Z, Gaston N, Newby GA, Xie J, Liu DR, Gao G, Wolfe SA, Khvorova A, Watts JK, Sontheimer EJ. Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo. bioRxiv. 2023 Mar 23. PMID: 36993169.
      Citations:    
    8. Hariharan VN, Shin M, Chang CW, O'Reilly D, Biscans A, Yamada K, Guo Z, Somasundaran M, Tang Q, Monopoli K, Krishnamurthy PM, Devi G, McHugh N, Cooper DA, Echeverria D, Cruz J, Chan IL, Liu P, Lim SY, McConnell J, Singh SP, Hildebrand S, Sousa J, Davis SM, Kennedy Z, Ferguson C, Godinho BMDC, Thillier Y, Caiazzi J, Ly S, Muhuri M, Kelly K, Humphries F, Cousineau A, Parsi KM, Li Q, Wang Y, Maehr R, Gao G, Korkin D, McDougall WM, Finberg RW, Fitzgerald KA, Wang JP, Watts JK, Khvorova A. Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection. Proc Natl Acad Sci U S A. 2023 03 14; 120(11):e2219523120. PMID: 36893269.
      Citations: 3     Fields:    Translation:HumansAnimalsCells
    9. Lin C, Yang Q, Guo D, Xie J, Yang YS, Chaugule S, DeSouza N, Oh WT, Li R, Chen Z, John AA, Qiu Q, Zhu LJ, Greenblatt MB, Ghosh S, Li S, Gao G, Haynes C, Emerson CP, Shim JH. Impaired mitochondrial oxidative metabolism in skeletal progenitor cells leads to musculoskeletal disintegration. Nat Commun. 2022 11 11; 13(1):6869. PMID: 36369293.
      Citations: 3     Fields:    Translation:AnimalsCells
    10. Namkung S, Tran NT, Manokaran S, He R, Su Q, Xie J, Gao G, Tai PWL. Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes. Hum Gene Ther. 2022 11; 33(21-22):1187-1196. PMID: 36178359.
      Citations: 2     Fields:    Translation:HumansCells
    11. Yang YS, Kim JM, Xie J, Chaugule S, Lin C, Ma H, Hsiao E, Hong J, Chun H, Shore EM, Kaplan FS, Gao G, Shim JH. Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery. Nat Commun. 2022 10 19; 13(1):6175. PMID: 36258013.
      Citations: 5     Fields:    Translation:HumansAnimalsCells
    12. Oh WT, Yang YS, Xie J, Ma H, Kim JM, Park KH, Oh DS, Park-Min KH, Greenblatt MB, Gao G, Shim JH. WNT-modulating gene silencers as a gene therapy for osteoporosis, bone fracture, and critical-sized bone defects. Mol Ther. 2023 02 01; 31(2):435-453. PMID: 36184851.
      Citations: 1     Fields:    Translation:Humans
    13. Tian B, Bilsbury E, Doherty S, Teebagy S, Wood E, Su W, Gao G, Lin H. Ocular Drug Delivery: Advancements and Innovations. Pharmaceutics. 2022 09 13; 14(9). PMID: 36145679.
      Citations:    
    14. Yenilmez B, Kelly M, Zhang GF, Wetoska N, Ilkayeva OR, Min K, Rowland L, DiMarzio C, He W, Raymond N, Lifshitz L, Pan M, Han X, Xie J, Friedline RH, Kim JK, Gao G, Herman MA, Newgard CB, Czech MP. Paradoxical activation of transcription factor SREBP1c and de novo lipogenesis by hepatocyte-selective ATP-citrate lyase depletion in obese mice. J Biol Chem. 2022 10; 298(10):102401. PMID: 35988648.
      Citations: 3     Fields:    Translation:AnimalsCells
    15. John AA, Xie J, Yang YS, Kim JM, Lin C, Ma H, Gao G, Shim JH. AAV-mediated delivery of osteoblast/osteoclast-regulating miRNAs for osteoporosis therapy. Mol Ther Nucleic Acids. 2022 Sep 13; 29:296-311. PMID: 35950212.
      Citations:    
    16. Zhang H, Bamidele N, Liu P, Ojelabi O, Gao XD, Rodriguez T, Cheng H, Kelly K, Watts JK, Xie J, Gao G, Wolfe SA, Xue W, Sontheimer EJ. Adenine Base Editing In Vivo with a Single Adeno-Associated Virus Vector. GEN Biotechnol. 2022 Jun 01; 1(3):285-299. PMID: 35811581.
      Citations:    
    17. Tran NT, Lecomte E, Saleun S, Namkung S, Robin C, Weber K, Devine E, Blouin V, Adjali O, Ayuso E, Gao G, Penaud-Budloo M, Tai PWL. Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity. Hum Gene Ther. 2022 04; 33(7-8):371-388. PMID: 35293222.
      Citations: 17     Fields:    Translation:HumansAnimalsCells
    18. 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 04; 604(7905):343-348. PMID: 35322228.
      Citations: 20     Fields:    Translation:HumansAnimalsCells
    19. Muhuri M, Levy DI, Schulz M, McCarty D, Gao G. Durability of transgene expression after rAAV gene therapy. Mol Ther. 2022 04 06; 30(4):1364-1380. PMID: 35283274.
      Citations: 9     Fields:    Translation:HumansAnimalsCells
    20. Kim JM, Yang YS, Xie J, Lee O, Kim J, Hong J, Boldyreff B, Filhol O, Chun H, Greenblatt MB, Gao G, Shim JH. Regulation of sclerostin by the SIRT1 stabilization pathway in osteocytes. Cell Death Differ. 2022 08; 29(8):1625-1638. PMID: 35169297.
      Citations: 6     Fields:    Translation:AnimalsCells
    21. Ibraheim R, Tai PWL, Mir A, Javeed N, Wang J, Rodr?guez 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 11 01; 12(1):6267. PMID: 34725353.
      Citations: 29     Fields:    Translation:HumansAnimalsCells
    22. Arjomandnejad M, Sylvia K, Blackwood M, Nixon T, Tang Q, Muhuri M, Gruntman AM, Gao G, Flotte TR, Keeler AM. Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells. Mol Ther Methods Clin Dev. 2021 Dec 10; 23:490-506. PMID: 34853797.
      Citations:    
    23. 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. 2022 01 05; 30(1):238-243. PMID: 34695545.
      Citations: 12     Fields:    Translation:Animals
    24. Cheng SY, Luo Y, Malachi A, Ko J, Su Q, Xie J, Tian B, Lin H, Ke X, Zheng Q, Tai PWL, Gao G, Punzo C. Low-Dose Recombinant Adeno-Associated Virus-Mediated Inhibition of Vascular Endothelial Growth Factor Can Treat Neovascular Pathologies Without Inducing Retinal Vasculitis. Hum Gene Ther. 2021 07; 32(13-14):649-666. PMID: 34182803.
      Citations: 2     Fields:    Translation:HumansAnimalsCells
    25. Zhan W, Muhuri M, Tai PWL, Gao G. Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens? Front Immunol. 2021; 12:673699. PMID: 34046041.
      Citations: 12     Fields:    Translation:HumansCells
    26. Muhuri M, Zhan W, Maeda Y, Li J, Lotun A, Chen J, Sylvia K, Dasgupta I, Arjomandnejad M, Nixon T, Keeler AM, Manokaran S, He R, Su Q, Tai PWL, Gao G. Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction. Front Immunol. 2021; 12:674242. PMID: 33995418.
      Citations: 9     Fields:    Translation:AnimalsCells
    27. Lotun A, Gessler DJ, Gao G. Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair. Front Cell Neurosci. 2021; 15:661928. PMID: 33967698.
      Citations:    
    28. Liu P, Liang SQ, Zheng C, Mintzer E, Zhao YG, Ponnienselvan K, Mir A, Sontheimer EJ, Gao G, Flotte TR, Wolfe SA, Xue W. Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice. Nat Commun. 2021 04 09; 12(1):2121. PMID: 33837189.
      Citations: 91     Fields:    Translation:HumansAnimalsCells
    29. Yang H, Brown RH, Wang D, Strauss KA, Gao G. AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies. Trends Mol Med. 2021 06; 27(6):520-523. PMID: 33714697.
      Citations: 5     Fields:    Translation:HumansCells
    30. Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021 02 08; 6(1):53. PMID: 33558455.
      Citations: 217     Fields:    Translation:HumansCells
    31. Han MS, Perry RJ, Camporez JP, Scherer PE, Shulman GI, Gao G, Davis RJ. A feed-forward regulatory loop in adipose tissue promotes signaling by the hepatokine FGF21. Genes Dev. 2021 01 01; 35(1-2):133-146. PMID: 33334822.
      Citations: 15     Fields:    Translation:AnimalsCells
    32. Tang Q, Keeler AM, Zhang S, Su Q, Lyu Z, Cheng Y, Gao G, Flotte TR. Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus. Biores Open Access. 2020; 9(1):219-228. PMID: 33117614.
      Citations:    
    33. Muhuri M, Gao G. Is smaller better? Vaccine targeting recombinant receptor-binding domain might hold the key for mass production of effective prophylactics to fight the COVID-19 pandemic. Signal Transduct Target Ther. 2020 10 06; 5(1):222. PMID: 33024086.
      Citations: 2     Fields:    Translation:HumansCellsPHPublic Health
    34. 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 Sep 11; 18:639-651. PMID: 32775498.
      Citations:    
    35. Hsu HL, Brown A, Loveland AB, Lotun A, Xu M, Luo L, Xu G, Li J, Ren L, Su Q, Gessler DJ, Wei Y, Tai PWL, Korostelev AA, Gao G. Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties. Nat Commun. 2020 06 30; 11(1):3279. PMID: 32606306.
      Citations: 21     Fields:    Translation:HumansAnimalsCells
    36. 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 Jun 12; 17:922-935. PMID: 32405514.
      Citations:    
    37. Wang D, Zhang F, Gao G. CRISPR-Based Therapeutic Genome Editing: Strategies and In?Vivo Delivery by AAV Vectors. Cell. 2020 04 02; 181(1):136-150. PMID: 32243786.
      Citations: 162     Fields:    Translation:HumansAnimalsCells
    38. 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. 2020 01 14; 52(1):167-182.e7. PMID: 31883839.
      Citations: 136     Fields:    Translation:HumansAnimalsCells
    39. 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. 2020 02 05; 28(2):422-430. PMID: 31843447.
      Citations: 11     Fields:    Translation:HumansAnimalsCells
    40. 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.
      Citations: 7     Fields:    
    41. 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. 2020 01; 45(2):384-393. PMID: 31277075.
      Citations: 13     Fields:    Translation:HumansAnimalsCells
    42. 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 07 04; 10(1):2958. PMID: 31273195.
      Citations: 44     Fields:    Translation:AnimalsCells
    43. Xu M, Li J, Xie J, He R, Su Q, Gao G, Tai PWL. High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs. Hum Gene Ther. 2019 08; 30(8):946-956. PMID: 31072208.
      Citations: 3     Fields:    Translation:HumansAnimalsCells
    44. Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019 05; 18(5):358-378. PMID: 30710128.
      Citations: 627     Fields:    Translation:HumansAnimalsCells
    45. 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.
      Citations: 11     Fields:    Translation:AnimalsCells
    46. 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 01; 29(1):23-27. PMID: 30640679.
      Citations: 7     Fields:    Translation:HumansCells
    47. 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 Dec 14; 11:65-72. PMID: 30397628.
      Citations:    
    48. 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 10; 36(9):839-842. PMID: 30102296.
      Citations: 22     Fields:    Translation:AnimalsCells
    49. 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 08; 29(8):853-860. PMID: 29597895.
      Citations: 29     Fields:    Translation:HumansAnimalsCells
    50. 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 04; 29(2):75-85. PMID: 29596011.
      Citations: 4     Fields:    Translation:HumansAnimalsCells
    51. 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 Jun 15; 9:234-246. PMID: 29766031.
      Citations:    
    52. Pfister EL, DiNardo N, Mondo E, Borel F, Conroy F, Fraser C, Gernoux G, Han X, Hu D, Johnson E, Kennington L, Liu P, Reid SJ, Sapp E, Vodicka P, Kuchel T, Morton AJ, Howland D, Moser R, Sena-Esteves M, Gao G, Mueller C, DiFiglia M, Aronin N. Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease. Hum Gene Ther. 2018 06; 29(6):663-673. PMID: 29207890.
      Citations: 43     Fields:    Translation:HumansAnimalsCells
    53. Tai PWL, Xie J, Fong K, Seetin M, Heiner C, Su Q, Weiand M, Wilmot D, Zapp ML, Gao G. Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras. Mol Ther Methods Clin Dev. 2018 Jun 15; 9:130-141. PMID: 29766023.
      Citations:    
    54. Wang D, Gao G. Taking a Hint from Structural Biology: To Better Understand AAV Transport across the BBB. Mol Ther. 2018 02 07; 26(2):336-338. PMID: 29398483.
      Citations: 1     Fields:    
    55. Yoon Y, Wang D, Tai PWL, Riley J, Gao G, Rivera-P?rez JA. Streamlined ex vivo and in vivo genome editing in mouse embryos using recombinant adeno-associated viruses. Nat Commun. 2018 01 29; 9(1):412. PMID: 29379011.
      Citations: 37     Fields:    Translation:HumansAnimalsCells
    56. Mondo E, Moser R, Gao G, Mueller C, Sena-Esteves M, Sapp E, Pfister E, O'Connell D, Takle K, Erger KE, Liu W, Conlon TJ, DiFiglia M, Gounis MJ, Aronin N. Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration. J Huntingtons Dis. 2018; 7(4):309-319. PMID: 30320596.
      Citations: 3     Fields:    Translation:HumansAnimalsCells
    57. 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 02; 10:349-360. PMID: 29499946.
      Citations:    
    58. Satishchandran A, Ambade A, Rao S, Hsueh YC, Iracheta-Vellve A, Tornai D, Lowe P, Gyongyosi B, Li J, Catalano D, Zhong L, Kodys K, Xie J, Bala S, Gao G, Szabo G. MicroRNA 122, Regulated by GRLH2, Protects Livers of Mice and?Patients From Ethanol-Induced Liver Disease. Gastroenterology. 2018 01; 154(1):238-252.e7. PMID: 28987423.
      Citations: 63     Fields:    Translation:HumansAnimalsCells
    59. Zhang W, Li L, Su Q, Gao G, Khanna H. Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis. Hum Gene Ther. 2018 01; 29(1):42-50. PMID: 28679290.
      Citations: 31     Fields:    Translation:HumansAnimalsCells
    60. Petit L, Ma S, Cheng SY, Gao G, Punzo C. Rod Outer Segment Development Influences AAV-Mediated Photoreceptor Transduction After Subretinal Injection. Hum Gene Ther. 2017 06; 28(6):464-481. PMID: 28510482.
      Citations: 13     Fields:    Translation:AnimalsCells
    61. 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 06 07; 25(6):1363-1374. PMID: 28462820.
      Citations: 44     Fields:    Translation:HumansAnimalsCells
    62. Mo C, Yang M, Han X, Li J, Gao G, Tai H, Huang N, Xiao H. Fat mass and obesity-associated protein attenuates lipid accumulation in macrophage foam cells and alleviates atherosclerosis in apolipoprotein E-deficient mice. J Hypertens. 2017 04; 35(4):810-821. PMID: 28253220.
      Citations: 19     Fields:    Translation:AnimalsCells
    63. Ji X, Saha S, Gao G, Lasek AW, Homanics GE, Guildford M, Tapper AR, Martin GE. The Sodium Channel ?4 Auxiliary Subunit Selectively Controls Long-Term Depression in Core Nucleus Accumbens Medium Spiny Neurons. Front Cell Neurosci. 2017; 11:17. PMID: 28243192.
      Citations:    
    64. GuhaSarkar D, Neiswender J, Su Q, Gao G, Sena-Esteves M. Intracranial AAV-IFN-? gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model. Mol Oncol. 2017 02; 11(2):180-193. PMID: 28098415.
      Citations: 20     Fields:    Translation:HumansAnimalsCells
    65. GuhaSarkar D, Su Q, Gao G, Sena-Esteves M. Systemic AAV9-IFN? gene delivery treats highly invasive glioblastoma. Neuro Oncol. 2016 11; 18(11):1508-1518. PMID: 27194146.
      Citations: 16     Fields:    Translation:HumansAnimalsCells
    66. 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 06; 24(6):1030-1041. PMID: 27039844.
      Citations: 15     Fields:    Translation:HumansAnimalsCells
    67. Gessler DJ, Gao G. Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders. Methods Mol Biol. 2016; 1382:429-65. PMID: 26611604.
      Citations: 11     Fields:    Translation:Humans
    68. Borel F, Gernoux G, Cardozo B, Metterville JP, Toro Cabrera GC, Song L, Su Q, Gao GP, Elmallah MK, Brown RH, Mueller C. Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates. Hum Gene Ther. 2016 01; 27(1):19-31. PMID: 26710998.
      Citations: 49     Fields:    Translation:HumansAnimalsCells
    69. 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 Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector. Mol Ther. 2016 Apr; 24(4):726-35. PMID: 26708003.
      Citations: 57     Fields:    Translation:AnimalsCells
    70. 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 01; 196(1):29-33. PMID: 26590313.
      Citations: 22     Fields:    Translation:AnimalsCells
    71. 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. PMID: 26526960.
      Citations: 7     Fields:    Translation:HumansAnimalsCells
    72. 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 Delivered Optimized AAV3B Vectors. Mol Ther. 2015 Dec; 23(12):1867-76. PMID: 26403887.
      Citations: 51     Fields:    Translation:HumansAnimalsCells
    73. 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. PMID: 26086867.
      Citations: 161     Fields:    Translation:HumansAnimalsCells
    74. Zhao-Shea R, DeGroot SR, Liu L, Vallaster M, Pang X, Su Q, Gao G, Rando OJ, Martin GE, George O, Gardner PD, Tapper AR. Increased CRF signalling in a ventral tegmental area-interpeduncular nucleus-medial habenula circuit induces anxiety during nicotine withdrawal. Nat Commun. 2015 Apr 21; 6:6770. PMID: 25898242.
      Citations: 75     Fields:    Translation:AnimalsCells
    75. 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. PMID: 25819833.
      Citations: 14     Fields:    Translation:HumansAnimalsCells
    76. Xie J, Burt DR, Gao G. Adeno-associated virus-mediated microRNA delivery and therapeutics. Semin Liver Dis. 2015 Feb; 35(1):81-8. PMID: 25632938.
      Citations: 16     Fields:    Translation:HumansCells
    77. Ahmed SS, 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. PMID: 25604619.
      Citations:    
    78. Ahmed SS, 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). PMID: 26550581.
      Citations:    
    79. Wang D, 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. PMID: 25227756.
      Citations: 36     Fields:    Translation:HumansCells
    80. Vernia S, Cavanagh-Kyros J, Garcia-Haro L, Sabio G, Barrett T, Jung DY, Kim JK, Xu J, Shulha HP, Garber M, Gao G, Davis RJ. The PPARa-FGF21 hormone axis contributes to metabolic regulation by the hepatic JNK signaling pathway. Cell Metab. 2014 Sep 02; 20(3):512-25. PMID: 25043817.
      Citations: 89     Fields:    Translation:AnimalsCells
    81. Wang D, Gao G. State-of-the-art human gene therapy: part I. Gene delivery technologies. Discov Med. 2014 Jul-Aug; 18(97):67-77. PMID: 25091489.
      Citations: 33     Fields:    Translation:Humans
    82. 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, Gao G. Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10. Mol Ther. 2014 Jul; 22(7):1299-1309. PMID: 24781136.
      Citations: 115     Fields:    Translation:AnimalsCells
    83. 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 Mar; 11(3):345-364. PMID: 24386892.
      Citations: 47     Fields:    Translation:HumansAnimalsCells
    84. Gao K, Li M, Zhong L, Su Q, Li J, Li S, He R, Zhang Y, Hendricks G, Wang J, 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. PMID: 25485285.
      Citations:    
    85. Pang X, Hogan EM, Casserly A, Gao G, Gardner PD, Tapper AR. Dicer expression is essential for adult midbrain dopaminergic neuron maintenance and survival. Mol Cell Neurosci. 2014 Jan; 58:22-8. PMID: 24184162.
      Citations: 14     Fields:    Translation:AnimalsCells
    86. Zhong L, Li S, Li M, Xie J, Zhang Y, Lee B, Batshaw ML, Wilson JM, Gao G. Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer. Hum Gene Ther. 2013 Sep; 24(9):814-9. PMID: 24010702.
      Citations: 2     Fields:    Translation:HumansCells
    87. 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 DA, Seyfried TN, Flotte TR, Matalon R, 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. PMID: 23817205.
      Citations: 51     Fields:    Translation:HumansAnimalsCells
    88. Jakovcevski M, Bharadwaj R, Straubhaar J, Gao G, Gavin DP, Jakovcevski I, Mitchell AC, Akbarian S. Prefrontal cortical dysfunction after overexpression of histone deacetylase 1. Biol Psychiatry. 2013 Nov 01; 74(9):696-705. PMID: 23664640.
      Citations: 25     Fields:    Translation:AnimalsCells
    89. Zhong L, Malani N, Li M, Brady T, Xie J, Bell P, Li S, Jones H, Wilson JM, Flotte TR, Bushman FD, 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. PMID: 23621841.
      Citations: 27     Fields:    Translation:Cells
    90. Ahmed SS, Gao G. Gene therapy for Canavan's disease takes a step forward. Mol Ther. 2013 Mar; 21(3):505-6. PMID: 23449107.
      Citations: 3     Fields:    Translation:HumansCells
    91. Velez G, Weingarden AR, Lei H, Kazlauskas A, Gao G. SU9518 inhibits proliferative vitreoretinopathy in fibroblast and genetically modified M?ller cell-induced rabbit models. Invest Ophthalmol Vis Sci. 2013 Feb 19; 54(2):1392-7. PMID: 23341018.
      Citations: 7     Fields:    Translation:HumansAnimalsCells
    92. Stoica L, Ahmed SS, Gao G, Sena-Esteves M. Gene transfer to the CNS using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14:Unit14D.5. PMID: 23686825.
      Citations: 12     Fields:    Translation:AnimalsCells
    93. Ahmed SS, Li J, Godwin J, Gao G, Zhong L. Gene transfer in the liver using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14:Unit14D.6. PMID: 23686826.
      Citations: 6     Fields:    Translation:AnimalsCells
    94. Venkatesh A, Ma S, Langellotto F, Gao G, Punzo C. Retinal gene delivery by rAAV and DNA electroporation. Curr Protoc Microbiol. 2013; Chapter 14:Unit 14D.4. PMID: 23408132.
      Citations: 20     Fields:    Translation:AnimalsCells
    95. Gruntman AM, Bish LT, Mueller C, Sweeney HL, Flotte TR, Gao G. Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14:Unit 14D.3. PMID: 23408131.
      Citations: 16     Fields:    Translation:AnimalsCells
    96. Gruntman AM, Mueller C, Flotte TR, Gao G. Gene transfer in the lung using recombinant adeno-associated virus. Curr Protoc Microbiol. 2012 Aug; Chapter 14:Unit14D.2. PMID: 22875566.
      Citations: 9     Fields:    Translation:AnimalsCells
    97. Mueller C, Ratner D, Zhong L, Esteves-Sena M, Gao G. Production and discovery of novel recombinant adeno-associated viral vectors. Curr Protoc Microbiol. 2012 Aug; Chapter 14:Unit14D.1. PMID: 22875565.
      Citations: 40     Fields:    Translation:HumansCells
    98. 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 04; 9(4):403-9. PMID: 22388288.
      Citations: 131     Fields:    Translation:AnimalsCells
    99. Gao G, Bish LT, Sleeper MM, Mu X, Sun L, Lou Y, Duan J, Hu C, Wang L, 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. PMID: 21563985.
      Citations: 29     Fields:    Translation:AnimalsCells
    100. Zhang H, Yang B, Mu X, Ahmed SS, Su Q, He R, Wang H, Mueller C, Sena-Esteves M, Brown R, Xu Z, 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. PMID: 21610699.
      Citations: 162     Fields:    Translation:AnimalsCells
    101. Gao G, Zhong L, Danos O. Exploiting natural diversity of AAV for the design of vectors with novel properties. Methods Mol Biol. 2011; 807:93-118. PMID: 22034027.
      Citations: 13     Fields:    Translation:HumansAnimalsCells
    102. 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, Gao G. MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. Mol Ther. 2011 Mar; 19(3):526-35. PMID: 21179009.
      Citations: 83     Fields:    Translation:HumansAnimalsCells
    103. Jakovcevski M, Guo Y, Su Q, Gao G, 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. PMID: 20360371.
      Citations: 3     Fields:    Translation:HumansAnimalsCells
    104. Zhang H, Xie J, Xie Q, Wilson JM, Gao G. Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production. Hum Gene Ther. 2009 Sep; 20(9):922-9. PMID: 19618999.
      Citations: 15     Fields:    Translation:HumansAnimalsCells
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