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Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6.
Mellen RW, Calabro KR, McCullough KT, Crosson SM, Cova A, Fajardo D, Xu E, Boye SL, Boye SE. Mellen RW, et al. Among authors: fajardo d. Mol Ther Methods Clin Dev. 2023 Jun 1;30:48-64. doi: 10.1016/j.omtm.2023.05.020. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37361352 Free PMC article.
Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque.
McCullough KT, Boye SL, Fajardo D, Calabro K, Peterson JJ, Strang CE, Chakraborty D, Gloskowski S, Haskett S, Samuelsson S, Jiang H, Witherspoon CD, Gamlin PD, Maeder ML, Boye SE. McCullough KT, et al. Among authors: fajardo d. Hum Gene Ther. 2019 May;30(5):571-589. doi: 10.1089/hum.2018.193. Epub 2018 Dec 20. Hum Gene Ther. 2019. PMID: 30358434 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. Among authors: fajardo d. 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.
Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis.
Boye SL, O'Riordan C, Morris J, Lukason M, Compton D, Baek R, Elmore DM, Peterson JJ, Fajardo D, McCullough KT, Scaria A, McVie-Wylie A, Boye SE. Boye SL, et al. Among authors: fajardo d. Mol Ther Methods Clin Dev. 2022 Dec 14;28:129-145. doi: 10.1016/j.omtm.2022.12.007. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2022. PMID: 36654798 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. Among authors: fajardo d. 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.
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.
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