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Guangping Gao PhD

TitleProfessor
Endowed TitlePenelope Booth Rockwell Chair in Biomedical Research 
InstitutionUniversity of Massachusetts Medical School
DepartmentMicrobiology and Physiological Systems
AddressUniversity of Massachusetts Medical School
368 Plantation Street, AS6-2049
Worcester MA 01605
Phone508-856-3563
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    Other Positions
    InstitutionUMMS - School of Medicine
    DepartmentGene Therapy Center

    InstitutionUMMS - School of Medicine
    DepartmentMicrobiology and Physiological Systems

    InstitutionUMMS - School of Medicine
    DepartmentNeuroNexus Institute

    InstitutionUMMS - School of Medicine
    DepartmentRNA Therapeutics Institute

    InstitutionUMMS - Graduate School of Biomedical Sciences
    DepartmentImmunology and Microbiology Program

    InstitutionUMMS - Graduate School of Biomedical Sciences
    DepartmentInterdisciplinary Graduate Program

    InstitutionUMMS - Graduate School of Biomedical Sciences
    DepartmentMD/PhD Program

    InstitutionUMMS - Graduate School of Biomedical Sciences
    DepartmentMillennium MD/PhD Program

    InstitutionUMMS - Graduate School of Biomedical Sciences
    DepartmentNeuroscience

    InstitutionUMMS - Graduate School of Biomedical Sciences
    DepartmentTranslational Science

    InstitutionUMMS - 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 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.
    List All   |   Timeline
    1. 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 rAAV-mediated inhibition of VEGF can treat neovascular pathologies without inducing retinal vasculitis. Hum Gene Ther. 2021 Jun 29. PMID: 34182803.
      View in: PubMed
    2. 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.
      View in: PubMed
    3. 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.
      View in: PubMed
    4. 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.
      View in: PubMed
    5. 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.
      View in: PubMed
    6. Yang H, Brown RH, Wang D, Strauss KA, Gao G. AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies. Trends Mol Med. 2021 Mar 10. PMID: 33714697.
      View in: PubMed
    7. Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021 Feb 08; 6(1):53. PMID: 33558455.
      View in: PubMed
    8. 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. 2020 Dec 17. PMID: 33334822.
      View in: PubMed
    9. 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.
      View in: PubMed
    10. 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.
      View in: PubMed
    11. 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.
      View in: PubMed
    12. 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 Jun 30; 11(1):3279. PMID: 32606306.
      View in: PubMed
    13. 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.
      View in: PubMed
    14. Wang D, Zhang F, Gao G. CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors. Cell. 2020 Apr 02; 181(1):136-150. PMID: 32243786.
      View in: PubMed
    15. 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.
      View in: PubMed
    16. 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.
      View in: PubMed
    17. 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.
      View in: PubMed
    18. 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.
      View in: PubMed
    19. 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.
      View in: PubMed
    20. 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.
      View in: PubMed
    21. 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.
      View in: PubMed
    22. 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.
      View in: PubMed
    23. 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.
      View in: PubMed
    24. 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.
      View in: PubMed
    25. 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 Sep 29. PMID: 30320596.
      View in: PubMed
    26. 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 Aug 13. PMID: 30102296.
      View in: PubMed
    27. 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 May 14. PMID: 29597895.
      View in: PubMed
    28. 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 Apr; 29(2):75-85. PMID: 29596011.
      View in: PubMed
    29. 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.
      View in: PubMed
    30. 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 Jun; 29(6):663-673. PMID: 29207890.
      View in: PubMed
    31. 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.
      View in: PubMed
    32. Wang D, Gao G. Taking a Hint from Structural Biology: To Better Understand AAV Transport across the BBB. Mol Ther. 2018 Feb 07; 26(2):336-338. PMID: 29398483.
      View in: PubMed
    33. 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 01 29; 9(1):412. PMID: 29379011.
      View in: PubMed
    34. 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.
      View in: PubMed
    35. 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.
      View in: PubMed
    36. 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 Jan; 29(1):42-50. PMID: 28679290.
      View in: PubMed
    37. 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 May 16. PMID: 28510482.
      View in: PubMed
    38. 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 07; 25(6):1363-1374. PMID: 28462820.
      View in: PubMed
    39. 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 Apr; 35(4):810-821. PMID: 28253220.
      View in: PubMed
    40. Ji X, Saha S, Gao G, Lasek AW, Homanics GE, Guildford M, Tapper AR, Martin GE. The Sodium Channel ß4 Auxiliary Subunit Selectively Controls Long-Term Depression in Core Nucleus Accumbens Medium Spiny Neurons. Front Cell Neurosci. 2017; 11:17. PMID: 28243192.
      View in: PubMed
    41. GuhaSarkar D, Neiswender J, Su Q, Gao G, Sena-Esteves M. Intracranial AAV-IFN-ß gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model. Mol Oncol. 2016 Nov 09. PMID: 28098415.
      View in: PubMed
    42. GuhaSarkar D, Su Q, Gao G, Sena-Esteves M. Systemic AAV9-IFNß gene delivery treats highly invasive glioblastoma. Neuro Oncol. 2016 Nov; 18(11):1508-1518. PMID: 27194146.
      View in: PubMed
    43. 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. PMID: 27039844.
      View in: PubMed
    44. Gessler DJ, Gao G. Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders. Methods Mol Biol. 2016; 1382:429-65. PMID: 26611604.
      View in: PubMed
    45. Borel F, Gernoux G, Cardozo B, Metterville JP, Toro Cabreja 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 Jan; 27(1):19-31. PMID: 26710998.
      View in: PubMed
    46. 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.
      View in: PubMed
    47. 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.
      View in: PubMed
    48. 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.
      View in: PubMed
    49. 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.
      View in: PubMed
    50. 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.
      View in: PubMed
    51. 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.
      View in: PubMed
    52. 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.
      View in: PubMed
    53. 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.
      View in: PubMed
    54. 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.
      View in: PubMed
    55. 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.
      View in: PubMed
    56. 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.
      View in: PubMed
    57. 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 2; 20(3):512-25. PMID: 25043817.
      View in: PubMed
    58. 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.
      View in: PubMed
    59. 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):1299-309. PMID: 24781136.
      View in: PubMed
    60. 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):345-64. PMID: 24386892.
      View in: PubMed
    61. 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.
      View in: PubMed
    62. 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.
      View in: PubMed
    63. 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.
      View in: PubMed
    64. 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.
      View in: PubMed
    65. 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 1; 74(9):696-705. PMID: 23664640.
      View in: PubMed
    66. 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.
      View in: PubMed
    67. Ahmed SS, Gao G. Gene therapy for Canavan's disease takes a step forward. Mol Ther. 2013 Mar; 21(3):505-6. PMID: 23449107.
      View in: PubMed
    68. Velez G, Weingarden AR, Lei H, Kazlauskas A, 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. PMID: 23341018.
      View in: PubMed
    69. 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.
      View in: PubMed
    70. 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.
      View in: PubMed
    71. 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.
      View in: PubMed
    72. 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.
      View in: PubMed
    73. 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.
      View in: PubMed
    74. 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.
      View in: PubMed
    75. 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.
      View in: PubMed
    76. 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.
      View in: PubMed
    77. 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.
      View in: PubMed
    78. 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.
      View in: PubMed
    79. 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.
      View in: PubMed
    80. 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.
      View in: PubMed
    81. 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.
      View in: PubMed
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