Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
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 …
mutations in the dystrophin gene. The majority of DMD mutations are deletions that …
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 …
male births and caused by mutations in the dystrophin gene. Genome editing has the …
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 …
children, is caused by the absence of dystrophin protein in striated muscle. Deletions of …
A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing
Y Zhang, H Li, T Nishiyama, JR McAnally… - … Therapy-Nucleic Acids, 2022 - cell.com
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by
mutations in the X-linked dystrophin (DMD) gene. Exon deletions flanking exon 51, which …
mutations in the X-linked dystrophin (DMD) gene. Exon deletions flanking exon 51, which …
CRISPR-mediated genome editing restores dystrophin expression and function in mdx mice
Duchenne muscular dystrophy (DMD) is a degenerative muscle disease caused by genetic
mutations that lead to the disruption of dystrophin in muscle fibers. There is no curative …
mutations that lead to the disruption of dystrophin in muscle fibers. There is no curative …
Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic
diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in …
diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in …
Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
DG Ousterout, AM Kabadi, PI Thakore… - Nature …, 2015 - nature.com
The CRISPR/Cas9 genome-editing platform is a promising technology to correct the genetic
basis of hereditary diseases. The versatility, efficiency and multiplexing capabilities of the …
basis of hereditary diseases. The versatility, efficiency and multiplexing capabilities of the …
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …
integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing …
Creation of a novel humanized dystrophic mouse model of duchenne muscular dystrophy and application of a CRISPR/Cas9 gene editing therapy
CS Young, E Mokhonova, M Quinonez… - Journal of …, 2017 - content.iospress.com
Duchenne muscular dystrophy is caused by mutations in DMD which disrupt the reading
frame. Therapeutic strategies that restore DMD's reading frame, such as exon skipping and …
frame. Therapeutic strategies that restore DMD's reading frame, such as exon skipping and …
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 …
characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete …