Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy
D Duan - Molecular Therapy, 2018 - cell.com
Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
JW McGreevy, CH Hakim… - Disease models & …, 2015 - journals.biologists.com
Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …
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 …
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …
Inhibition of prostaglandin-degrading enzyme 15-PGDH rejuvenates aged muscle mass and strength
INTRODUCTION Currently there are no approved treatments for sarcopenia, the age-
dependent loss of skeletal muscle mass and strength that constitutes a major public health …
dependent loss of skeletal muscle mass and strength that constitutes a major public health …
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 …
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 …
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 …
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease
caused by mutations in the dystrophin gene. Gene therapy using highly functional …
caused by mutations in the dystrophin gene. Gene therapy using highly functional …
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by
the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene …
the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene …
Drug development progress in duchenne muscular dystrophy
J Deng, J Zhang, K Shi, Z Liu - Frontiers in Pharmacology, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe, progressive, and incurable X-linked
disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of …
disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of …