Antisense Oligonucleotide-Based Splice Correction of a Deep-Intronic Mutation in CHM Underlying Choroideremia

Garanto, A; Van der Velde-Visser, SD; Cremers, FPM; Collin, RWJ

Garanto, A (reprint author), Radboud Univ Nijmegen, Med Ctr, Dept Human Genet, Nijmegen, Netherlands.; Garanto, A (reprint author), Radboud Univ Nijmegen, Med Ctr, Donders Inst Brain Cognit & Behav, Nijmegen, Netherlands.

RETINAL DEGENERATIVE DISEASES: MECHANISMS AND EXPERIMENTAL THERAPY, 2018; 1074 (): 83

Abstract

Choroideremia is a progressive genetic eye disorder caused by mutations in the CHM gene that encodes the Rab escort protein-1 (REP-1). One of the many......

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