Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA

Dooley, SJ; McDougald, DS; Fisher, KJ; Bennicelli, JL; Mitchell, LG; Bennett, J

Bennett, J (reprint author), Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Perelman Sch Med, Dept Ophthalmol, 310 Stellar Chance Labs,422 Curie Blvd, Philadelphia, PA 19104 USA.

MOLECULAR THERAPY-NUCLEIC ACIDS, 2018; 12 (): 294

Abstract

Ocular gene therapy with recombinant adeno-associated virus (AAV) has shown vector-mediated gene augmentation to be safe and efficacious in the retina......

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