Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

A Moretti, L Fonteyne, F Giesert, P Hoppmann… - Nature medicine, 2020 - nature.com
Frameshift mutations in the DMD gene, encoding dystrophin, cause Duchenne muscular
dystrophy (DMD), leading to terminal muscle and heart failure in patients. Somatic gene …

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

M Tabebordbar, K Zhu, JKW Cheng, WL Chew… - Science, 2016 - science.org
Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …

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

L Amoasii, C Long, H Li, AA Mireault… - Science translational …, 2017 - science.org
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle disease caused by
mutations in the dystrophin gene. The majority of DMD mutations are deletions that …

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

F Chemello, AC Chai, H Li, C Rodriguez-Caycedo… - Science …, 2021 - science.org
Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …

Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy

JN Robinson-Hamm, CA Gersbach - Human genetics, 2016 - Springer
Duchenne muscular dystrophy is one of the most common inherited genetic diseases and is
caused by mutations to the DMD gene that encodes the dystrophin protein. Recent …

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

CE Nelson, CH Hakim, DG Ousterout, PI Thakore… - Science, 2016 - science.org
Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000
male births and caused by mutations in the dystrophin gene. Genome editing has the …

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

YL Min, H Li, C Rodriguez-Caycedo, AA Mireault… - Science …, 2019 - science.org
Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is
characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete …

[HTML][HTML] Correction of three prominent mutations in mouse and human models of Duchenne muscular dystrophy by single-cut genome editing

YL Min, F Chemello, H Li, C Rodriguez-Caycedo… - Molecular Therapy, 2020 - cell.com
Duchenne muscular dystrophy (DMD), one of the most common neuromuscular disorders of
children, is caused by the absence of dystrophin protein in striated muscle. Deletions of …

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

L Amoasii, JCW Hildyard, H Li, E Sanchez-Ortiz… - Science, 2018 - science.org
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

CE Nelson, Y Wu, MP Gemberling, ML Oliver… - Nature medicine, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for
therapeutic genome editing. There have been several recent reports of genome editing in …