Open AccessAssessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy
Author(s) -
Jerry R. Mendell,
Zarife Sahenk,
Kelly J. Lehman,
C. Nease,
Linda Lowes,
N. Miller,
M. Iammarino,
Lindsay N. Alfano,
Amanda Nicholl,
Samiah Al-Zaidy,
Sarah Lewis,
Kathleen Church,
Richard Shell,
Linda Cripe,
Rachael A. Potter,
Danielle A. Griffin,
Eric R. Pozsgai,
A. Dugar,
Mark Hogan,
Louise R. RodinoKlapac
Publication year - 2020
Publication title -
jama neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 5.298
H-Index - 231
eISSN - 2168-6157
pISSN - 2168-6149
DOI - 10.1001/jamaneurol.2020.1484
Subject(s) - medicine , duchenne muscular dystrophy , dystrophin , tolerability , adverse effect , pediatrics
Micro-dystrophin gene transfer shows promise for treating patients with Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype rh74 (rAAVrh74) and codon-optimized human micro-dystrophin driven by a skeletal and cardiac muscle-specific promoter with enhanced cardiac expression (MHCK7).
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