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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: elmore dm. 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.