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21 results

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Page 1
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results.
Flotte TR, Trapnell BC, Humphries M, Carey B, Calcedo R, Rouhani F, Campbell-Thompson M, Yachnis AT, Sandhaus RA, McElvaney NG, Mueller C, Messina LM, Wilson JM, Brantly M, Knop DR, Ye GJ, Chulay JD. Flotte TR, et al. Among authors: knop dr. Hum Gene Ther. 2011 Oct;22(10):1239-47. doi: 10.1089/hum.2011.053. Epub 2011 Aug 24. Hum Gene Ther. 2011. PMID: 21609134 Free PMC article. Clinical Trial.
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression.
Mueller C, Chulay JD, Trapnell BC, Humphries M, Carey B, Sandhaus RA, McElvaney NG, Messina L, Tang Q, Rouhani FN, Campbell-Thompson M, Fu AD, Yachnis A, Knop DR, Ye GJ, Brantly M, Calcedo R, Somanathan S, Richman LP, Vonderheide RH, Hulme MA, Brusko TM, Wilson JM, Flotte TR. Mueller C, et al. Among authors: knop dr. J Clin Invest. 2013 Dec;123(12):5310-8. doi: 10.1172/JCI70314. Epub 2013 Nov 15. J Clin Invest. 2013. PMID: 24231351 Free PMC article. Clinical Trial.
Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin.
Ye GJ, Budzynski E, Sonnentag P, Miller PE, Sharma AK, Ver Hoeve JN, Howard K, Knop DR, Neuringer M, McGill T, Stoddard J, Chulay JD. Ye GJ, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2015 Sep;26(3):165-76. doi: 10.1089/humc.2015.076. Hum Gene Ther Clin Dev. 2015. PMID: 26390090 Free PMC article.
Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.
Ye GJ, Budzynski E, Sonnentag P, Nork TM, Miller PE, McPherson L, Ver Hoeve JN, Smith LM, Arndt T, Mandapati S, Robinson PM, Calcedo R, Knop DR, Hauswirth WW, Chulay JD. Ye GJ, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2016 Mar;27(1):27-36. doi: 10.1089/humc.2015.163. Hum Gene Ther Clin Dev. 2016. PMID: 27003752 Free PMC article.
Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.
Ye GJ, Budzynski E, Sonnentag P, Nork TM, Miller PE, Sharma AK, Ver Hoeve JN, Smith LM, Arndt T, Calcedo R, Gaskin C, Robinson PM, Knop DR, Hauswirth WW, Chulay JD. Ye GJ, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2016 Mar;27(1):37-48. doi: 10.1089/humc.2015.164. Hum Gene Ther Clin Dev. 2016. PMID: 27003753 Free PMC article.
Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep.
Gootwine E, Ofri R, Banin E, Obolensky A, Averbukh E, Ezra-Elia R, Ross M, Honig H, Rosov A, Yamin E, Ye GJ, Knop DR, Robinson PM, Chulay JD, Shearman MS. Gootwine E, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2017 Jun;28(2):96-107. doi: 10.1089/humc.2017.028. Epub 2017 May 5. Hum Gene Ther Clin Dev. 2017. PMID: 28478700
Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Song C, Conlon TJ, Deng WT, Coleman KE, Zhu P, Plummer C, Mandapati S, Van Hoosear M, Green KB, Sonnentag P, Sharma AK, Timmers A, Robinson PM, Knop DR, Hauswirth WW, Chulay JD, Shearman MS, Ye GJ. Song C, et al. Among authors: knop dr. Hum Gene Ther Clin Dev. 2018 Dec;29(4):188-197. doi: 10.1089/humc.2018.168. Hum Gene Ther Clin Dev. 2018. PMID: 30280954 Free PMC article.
21 results