[HTML][HTML] A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation
Duchenne muscular dystrophy (DMD), caused by mutations in the X-linked dystrophin gene,
is a lethal neuromuscular disease. Correction of DMD mutations in animal models has been …
is a lethal neuromuscular disease. Correction of DMD mutations in animal models has been …
[HTML][HTML] AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice
Adeno-associated virus–mediated (AAV-mediated) CRISPR editing is a revolutionary
approach for treating inherited diseases. Sustained, often life-long mutation correction is …
approach for treating inherited diseases. Sustained, often life-long mutation correction is …
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 …
dystrophy (DMD), leading to terminal muscle and heart failure in patients. Somatic gene …
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 …
Genome editing for Duchenne muscular dystrophy: a glimpse of the future?
Mutations in Dystrophin, one of the largest proteins in the mammalian body, are causative
for a severe form of muscle disease, Duchenne Muscular Dystrophy (DMD), affecting not …
for a severe form of muscle disease, Duchenne Muscular Dystrophy (DMD), affecting not …
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 …
Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by
mutations in the dystrophin gene (DMD). Previously, we applied CRISPR-Cas9–mediated …
mutations in the dystrophin gene (DMD). Previously, we applied CRISPR-Cas9–mediated …
CRISPR-based therapeutic gene editing for Duchenne muscular dystrophy: advances, challenges and perspectives
G Chen, T Wei, H Yang, G Li, H Li - Cells, 2022 - mdpi.com
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-
of-function mutations in the dystrophin gene and characterized by progressive muscle …
of-function mutations in the dystrophin gene and characterized by progressive muscle …
CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
Duchenne muscular dystrophy (DMD), caused by mutations in the X-linked dystrophin gene
(DMD), is characterized by fatal degeneration of striated muscles. Dilated cardiomyopathy is …
(DMD), is characterized by fatal degeneration of striated muscles. Dilated cardiomyopathy is …
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 …