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Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.
Beltran WA, Cideciyan AV, Boye SE, Ye GJ, Iwabe S, Dufour VL, Marinho LF, Swider M, Kosyk MS, Sha J, Boye SL, Peterson JJ, Witherspoon CD, Alexander JJ, Ying GS, Shearman MS, Chulay JD, Hauswirth WW, Gamlin PD, Jacobson SG, Aguirre GD. Beltran WA, et al. Among authors: dufour vl. Mol Ther. 2017 Aug 2;25(8):1866-1880. doi: 10.1016/j.ymthe.2017.05.004. Epub 2017 May 27. Mol Ther. 2017. PMID: 28566226 Free PMC article.
BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure.
Guziewicz KE, Cideciyan AV, Beltran WA, Komáromy AM, Dufour VL, Swider M, Iwabe S, Sumaroka A, Kendrick BT, Ruthel G, Chiodo VA, Héon E, Hauswirth WW, Jacobson SG, Aguirre GD. Guziewicz KE, et al. Among authors: dufour vl. Proc Natl Acad Sci U S A. 2018 Mar 20;115(12):E2839-E2848. doi: 10.1073/pnas.1720662115. Epub 2018 Mar 5. Proc Natl Acad Sci U S A. 2018. PMID: 29507198 Free PMC article.
Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector.
Cideciyan AV, Sudharsan R, Dufour VL, Massengill MT, Iwabe S, Swider M, Lisi B, Sumaroka A, Marinho LF, Appelbaum T, Rossmiller B, Hauswirth WW, Jacobson SG, Lewin AS, Aguirre GD, Beltran WA. Cideciyan AV, et al. Among authors: dufour vl. Proc Natl Acad Sci U S A. 2018 Sep 4;115(36):E8547-E8556. doi: 10.1073/pnas.1805055115. Epub 2018 Aug 20. Proc Natl Acad Sci U S A. 2018. PMID: 30127005 Free PMC article.
Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy.
Gardiner KL, Cideciyan AV, Swider M, Dufour VL, Sumaroka A, Komáromy AM, Hauswirth WW, Iwabe S, Jacobson SG, Beltran WA, Aguirre GD. Gardiner KL, et al. Among authors: dufour vl. Mol Ther. 2020 Jan 8;28(1):266-278. doi: 10.1016/j.ymthe.2019.08.013. Epub 2019 Sep 3. Mol Ther. 2020. PMID: 31604676 Free PMC article.
Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa.
Dufour VL, Cideciyan AV, Ye GJ, Song C, Timmers A, Habecker PL, Pan W, Weinstein NM, Swider M, Durham AC, Ying GS, Robinson PM, Jacobson SG, Knop DR, Chulay JD, Shearman MS, Aguirre GD, Beltran WA. Dufour VL, et al. Hum Gene Ther. 2020 Feb;31(3-4):253-267. doi: 10.1089/hum.2019.297. Epub 2020 Feb 6. Hum Gene Ther. 2020. PMID: 31910043 Free PMC article.
Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy.
Song C, Dufour VL, Cideciyan AV, Ye GJ, Swider M, Newmark JA, Timmers AM, Robinson PM, Knop DR, Chulay JD, Jacobson SG, Aguirre GD, Beltran WA, Shearman MS. Song C, et al. Among authors: dufour vl. Hum Gene Ther. 2020 Jul;31(13-14):743-755. doi: 10.1089/hum.2019.337. Epub 2020 Jun 29. Hum Gene Ther. 2020. PMID: 32414297 Free PMC article.
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