Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models

Barthelemy, F; Wang, RT; Hsu, C; Douine, ED; Marcantonio, EE; Nelson, SF; Miceli, MC

Miceli, MC (reprint author), Univ Calif Los Angeles, David Geffen Sch Med, Dept Microbiol Immunol & Mol Genet, 277B Biomed Sci Res Bldg,615 Charles E Young Dr, Los Angeles, CA 90095 USA.; Miceli, MC (reprint author), Univ Calif Los Angeles, Coll Letters &

MOLECULAR THERAPY-NUCLEIC ACIDS, 2019; 18 (): 580

Abstract

Systemic delivery of antisense oligonucleotides (AO) for DMD exon skipping has proven effective for reframing DMD mRNA, rescuing dystrophin expression......

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