Efrain Sanchez‐Ortiz

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

Chengzu Long, Leonela Amoasii, Alex A. Mireault et al. · Science · 2015 · 926 citations

Engineering, Genetics, Genomic Mechanism +17

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

Leonela Amoasii, John Hildyard, Hui Li et al. · Science · 2018 · 533 citations · Full text

Control of muscle formation by the fusogenic micropeptide myomixer

Pengpeng Bi, Andres Ramirez-Martinez, Hui Li et al. · Science · 2017 · 398 citations · Full text

Mechanobiology, Myomixer-myomaker Pair, Muscle Function +14

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

Leonela Amoasii, Chengzu Long, Hui Li et al. · Science Translational Medicine · 2017 · 243 citations · Full text

Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing

Francesco Chemello, Andreas C. Chai, Hui Li et al. · Science Advances · 2021 · 224 citations · Full text

In Vivo Gene Therapy, Genetics, Molecular Biology +27