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 …
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 …
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 …
Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice
Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by mutations in the
dystrophin gene. CRISPR/Cas9 genome editing has been used to correct DMD mutations in …
dystrophin gene. CRISPR/Cas9 genome editing has been used to correct DMD 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 …
[HTML][HTML] Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy
A Pickar-Oliver, V Gough, JD Bohning, S Liu… - Molecular Therapy, 2021 - cell.com
Targeted gene-editing strategies have emerged as promising therapeutic approaches for
the permanent treatment of inherited genetic diseases. However, precise gene correction …
the permanent treatment of inherited genetic diseases. However, precise gene correction …
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 …
caused by mutations to the DMD gene that encodes the dystrophin protein. Recent …
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 …
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 …