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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: peterson jj. 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.
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: peterson jj. 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.
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: peterson jj. 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.
167 results