Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6

Mellen, RW; Calabro, KR; Mccullough, KT; Crosson, SM; de la Cova, A; Fajardo, D; Xu, EMY; Boye, SL; Boye, SE

Boye, SE (通讯作者)，Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, POB 100296, Gainesville, FL 32610 USA.

MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2023; 30 (): 48

Abstract

Cone-rod dystrophy 6 (CORD6) is caused by gain-of-function mutations in the GUCY2D gene, which encodes retinal guanylate cyclase-1 (RetGC1). There are......

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