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Large1 Gene Transfer in Older myd Mice with Severe Muscular Dystrophy Restores Muscle Function and Greatly Improves Survival
Preprint
Overview
Research
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Overview
cited authors
Yonekawa, Takahiro; Rauckhorst, Adam J; El-Hattab, Sara; Cuellar, Marco A; Venzke, David; Anderson, Mary E; Okuma, Hidehiko; Pewa, Alvin D; Taylor, Eric B; Campbell, Kevin P
authors
Rauckhorst, Adam
Research
keywords
2.1 Biological and endogenous factors
32 Biomedical and Clinical Sciences
3208 Medical Physiology
5.1 Pharmaceuticals
Biotechnology
Brain Disorders
Congenital Muscular Dystrophy
Congenital Structural Anomalies
Duchenne/ Becker Muscular Dystrophy
Gene Therapy
Genetics
Intellectual and Developmental Disabilities (IDD)
Muscular Dystrophy
Musculoskeletal
Neurosciences
Orphan Drug
Pediatric
Pediatric Research Initiative
Rare Diseases
Identifiers
Digital Object Identifier (DOI)
https://doi.org/10.1101/2021.10.28.466309