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Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.
Beltran WA, Cideciyan AV, Lewin AS, Iwabe S, Khanna H, Sumaroka A, Chiodo VA, Fajardo DS, Román AJ, Deng WT, Swider M, Alemán TS, Boye SL, Genini S, Swaroop A, Hauswirth WW, Jacobson SG, Aguirre GD. Beltran WA, et al. Among authors: fajardo ds. Proc Natl Acad Sci U S A. 2012 Feb 7;109(6):2132-7. doi: 10.1073/pnas.1118847109. Epub 2012 Jan 23. Proc Natl Acad Sci U S A. 2012. PMID: 22308428 Free PMC article.
Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy.
Boye SL, Choudhury S, Crosson S, Di Pasquale G, Afione S, Mellen R, Makal V, Calabro KR, Fajardo D, Peterson J, Zhang H, Leahy MT, Jennings CK, Chiorini JA, Boyd RF, Boye SE. Boye SL, et al. Mol Ther. 2020 Jun 3;28(6):1464-1478. doi: 10.1016/j.ymthe.2020.04.002. Epub 2020 Apr 11. Mol Ther. 2020. PMID: 32304666 Free PMC article.
Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion.
Kellish PC, Marsic D, Crosson SM, Choudhury S, Scalabrino ML, Strang CE, Hill J, McCullough KT, Peterson JJ, Fajardo D, Gupte S, Makal V, Kondratov O, Kondratova L, Iyer S, Witherspoon CD, Gamlin PD, Zolotukhin S, Boye SL, Boye SE. Kellish PC, et al. Mol Ther. 2023 Dec 6;31(12):3441-3456. doi: 10.1016/j.ymthe.2023.10.001. Epub 2023 Oct 9. Mol Ther. 2023. PMID: 37814449 Free article.
13 results