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Guangping Gao to Dependovirus

This is a "connection" page, showing publications Guangping Gao has written about Dependovirus.
Connection Strength

14.925
  1. Keeler AM, Zhan W, Ram S, Fitzgerald KA, Gao G. The curious case of AAV immunology. Mol Ther. 2025 May 07; 33(5):1946-1965.
    View in: PubMed
    Score: 0.589
  2. Wang JH, Zhan W, Gallagher TL, Gao G. Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review. Mol Ther. 2024 Dec 04; 32(12):4185-4207.
    View in: PubMed
    Score: 0.572
  3. Wang JH, Gessler DJ, Zhan W, Gallagher TL, Gao G. Adeno-associated virus as a delivery vector for gene therapy of human diseases. Signal Transduct Target Ther. 2024 04 03; 9(1):78.
    View in: PubMed
    Score: 0.550
  4. 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.
    View in: PubMed
    Score: 0.477
  5. 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.
    View in: PubMed
    Score: 0.450
  6. 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.
    View in: PubMed
    Score: 0.449
  7. Qin W, Xu G, Tai PWL, Wang C, Luo L, Li C, Hu X, Xue J, Lu Y, Zhou Q, Wei Q, Wen T, Hu J, Xiao Y, Yang L, Li W, Flotte TR, Wei Y, Gao G. Large-scale molecular epidemiological analysis of AAV in a cancer patient population. Oncogene. 2021 Apr; 40(17):3060-3071.
    View in: PubMed
    Score: 0.446
  8. 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.
    View in: PubMed
    Score: 0.445
  9. 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.
    View in: PubMed
    Score: 0.442
  10. 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.
    View in: PubMed
    Score: 0.424
  11. 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.
    View in: PubMed
    Score: 0.417
  12. 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.
    View in: PubMed
    Score: 0.407
  13. 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.
    View in: PubMed
    Score: 0.391
  14. 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.
    View in: PubMed
    Score: 0.382
  15. 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.
    View in: PubMed
    Score: 0.363
  16. 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.
    View in: PubMed
    Score: 0.340
  17. 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.
    View in: PubMed
    Score: 0.310
  18. 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.
    View in: PubMed
    Score: 0.305
  19. 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.
    View in: PubMed
    Score: 0.276
  20. 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.
    View in: PubMed
    Score: 0.270
  21. 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.
    View in: PubMed
    Score: 0.264
  22. 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.
    View in: PubMed
    Score: 0.261
  23. 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.
    View in: PubMed
    Score: 0.258
  24. Ahmed SS, Gao G. Gene therapy for Canavan's disease takes a step forward. Mol Ther. 2013 Mar; 21(3):505-6.
    View in: PubMed
    Score: 0.255
  25. 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.
    View in: PubMed
    Score: 0.252
  26. 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.
    View in: PubMed
    Score: 0.245
  27. 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.
    View in: PubMed
    Score: 0.245
  28. 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.
    View in: PubMed
    Score: 0.238
  29. 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.
    View in: PubMed
    Score: 0.227
  30. 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.
    View in: PubMed
    Score: 0.226
  31. 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.
    View in: PubMed
    Score: 0.220
  32. 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.
    View in: PubMed
    Score: 0.219
  33. 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.
    View in: PubMed
    Score: 0.200
  34. Xing R, Xu M, Reil D, Destefano A, Cui M, Liu N, Liang J, Xu G, Luo L, Xu M, Zhang F, Tai PWL, Wei Y, Gruntman AM, Flotte TR, Gao G, Wang D. A single amino acid variant in the variable region I of AAV capsid confers liver detargeting. PLoS Pathog. 2025 Sep; 21(9):e1013533.
    View in: PubMed
    Score: 0.152
  35. Liu H, Liu N, Zhou C, Du A, Kapadia M, Tai PWL, Barton E, Gao G, Wang D. High-purity AAV vector production utilizing recombination-dependent minicircle formation and genetic coupling. EMBO Mol Med. 2025 Jun; 17(6):1475-1494.
    View in: PubMed
    Score: 0.149
  36. Du A, Yang K, Zhou X, Ren L, Liu N, Zhou C, Liang J, Yan N, Gao G, Wang D. Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency. JCI Insight. 2024 Oct 08; 9(19).
    View in: PubMed
    Score: 0.143
  37. Cui M, Su Q, Yip M, McGowan J, Punzo C, Gao G, Tai PWL. The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2. Gene Ther. 2024 Sep; 31(9-10):489-498.
    View in: PubMed
    Score: 0.141
  38. 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 10; 35(19-20):814-824.
    View in: PubMed
    Score: 0.140
  39. Wang J, Gao G, Wang D. Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders. Neurotherapeutics. 2024 Jul; 21(4):e00391.
    View in: PubMed
    Score: 0.140
  40. Lin C, Yang YS, Ma H, Chen Z, Chen D, John AA, Xie J, Gao G, Shim JH. Engineering a targeted and safe bone anabolic gene therapy to treat osteoporosis in alveolar bone loss. Mol Ther. 2024 Sep 04; 32(9):3080-3100.
    View in: PubMed
    Score: 0.140
  41. Sato T, Chaugule S, Greenblatt MB, Gao G, Shim JH. Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus. Hum Gene Ther. 2024 05; 35(9-10):329-341.
    View in: PubMed
    Score: 0.138
  42. Lin C, Greenblatt MB, Gao G, Shim JH. Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases. Hum Gene Ther. 2024 05; 35(9-10):317-328.
    View in: PubMed
    Score: 0.138
  43. 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).
    View in: PubMed
    Score: 0.130
  44. 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.
    View in: PubMed
    Score: 0.120
  45. 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.
    View in: PubMed
    Score: 0.119
  46. 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.
    View in: PubMed
    Score: 0.116
  47. 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.
    View in: PubMed
    Score: 0.099
  48. 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.
    View in: PubMed
    Score: 0.099
  49. Sanmiguel J, Gao G, Vandenberghe LH. Quantitative and Digital Droplet-Based AAV Genome Titration. Methods Mol Biol. 2019; 1950:51-83.
    View in: PubMed
    Score: 0.096
  50. 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.
    View in: PubMed
    Score: 0.093
  51. 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.
    View in: PubMed
    Score: 0.090
  52. 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.
    View in: PubMed
    Score: 0.089
  53. 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.
    View in: PubMed
    Score: 0.086
  54. 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.
    View in: PubMed
    Score: 0.086
  55. 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.
    View in: PubMed
    Score: 0.083
  56. 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.
    View in: PubMed
    Score: 0.079
  57. 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 07; 53(5):3235-3248.
    View in: PubMed
    Score: 0.075
  58. 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.
    View in: PubMed
    Score: 0.074
  59. Xie J, Burt DR, Gao G. Adeno-associated virus-mediated microRNA delivery and therapeutics. Semin Liver Dis. 2015 Feb; 35(1):81-8.
    View in: PubMed
    Score: 0.073
  60. 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.
    View in: PubMed
    Score: 0.063
  61. Stoica L, Ahmed SS, Gao G, Sena-Esteves M. Gene transfer to the CNS using recombinant adeno-associated virus. Curr Protoc Microbiol. 2013; Chapter 14:14D.5.1-14D.5.18.
    View in: PubMed
    Score: 0.063
  62. 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 14:14D.6.1-14D.6.32.
    View in: PubMed
    Score: 0.063
  63. 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.
    View in: PubMed
    Score: 0.052
  64. Bamidele N, Pires-Ferreira D, Zheng C, Destefano A, Cheng H, Tang Q, Cao Y, Xie J, Gao G, Gruntman A, Sontheimer E, Flotte TR, Xue W. A compact base editor rescues AATD-associated liver and lung disease in mouse models. Mol Ther. 2025 Nov 05; 33(11):5817-5828.
    View in: PubMed
    Score: 0.038
  65. Wang Y, Liu J, Zhu S, Hu S, Chen X, Mandon E, Tran NT, Zhang S, Qi Y, Ma H, He R, Cao Y, Su Q, Gallagher TL, Li Z, Zhou C, Tai PWL, Gao G, Xie J. miR-375 protects against acetaminophen-induced acute liver failure by orchestrating pharmacogene expression. Mol Ther. 2025 Oct 01; 33(10):4874-4888.
    View in: PubMed
    Score: 0.037
  66. Wang J, Poskitt LE, Gallagher J, Puffenberger EG, Wynn RM, Shishodia G, Chuang DT, Beever J, Hardin DL, Brigatti KW, Baker WC, Gately R, Bertrand S, Rodrigues A, Benatti HR, Taghian T, Hall E, Prestigiacomo R, Liang J, Chen G, Zhou X, Ren L, Liu N, He R, Su Q, Xie J, Jiang Z, Gruntman A, Gray-Edwards H, Gao G, Strauss KA, Wang D. BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease. Sci Transl Med. 2025 Feb 26; 17(787):eads0539.
    View in: PubMed
    Score: 0.037
  67. 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).
    View in: PubMed
    Score: 0.033
  68. 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.
    View in: PubMed
    Score: 0.031
  69. 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.
    View in: PubMed
    Score: 0.028
  70. 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.
    View in: PubMed
    Score: 0.028
  71. Xiao W, Gao G, Ling C, Herzog RW, Xiao X, Samulski RJ. Impact of neutralizing antibodies against AAV is a key consideration in gene transfer to nonhuman primates. Nat Med. 2018 06; 24(6):699.
    View in: PubMed
    Score: 0.023
  72. 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.
    View in: PubMed
    Score: 0.023
  73. 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.
    View in: PubMed
    Score: 0.023
  74. 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 Dec 04; 365:192-205.
    View in: PubMed
    Score: 0.022
  75. 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.
    View in: PubMed
    Score: 0.020
  76. 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.
    View in: PubMed
    Score: 0.019
  77. 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.
    View in: PubMed
    Score: 0.019
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