Dystrophin News and Research

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Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.
New CRISPR gene-editing technique can correct majority of mutations that cause DMD

New CRISPR gene-editing technique can correct majority of mutations that cause DMD

UT Southwestern researchers develop improved gene-editing technique to correct DMD mutations

UT Southwestern researchers develop improved gene-editing technique to correct DMD mutations

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

Study identifies predictors of poor outcomes in Duchenne muscular dystrophy patients

Study identifies predictors of poor outcomes in Duchenne muscular dystrophy patients

CRISPR-Gold corrects gene mutations in mice with Duchenne muscular dystrophy

CRISPR-Gold corrects gene mutations in mice with Duchenne muscular dystrophy

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Researchers discover connection between processes that keep the heart from repairing itself

Researchers discover connection between processes that keep the heart from repairing itself

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Researchers correct Duchenne muscular dystrophy using gene-editing alternative

Researchers correct Duchenne muscular dystrophy using gene-editing alternative

Early treatment with heart failure drug can improve cardiac function in young boys with DMD

Early treatment with heart failure drug can improve cardiac function in young boys with DMD

FDA approves new treatment for wide range of patients with Duchenne muscular dystrophy

FDA approves new treatment for wide range of patients with Duchenne muscular dystrophy

FDA approval of controversial drug for Duchenne muscular dystrophy raises concern and hope

FDA approval of controversial drug for Duchenne muscular dystrophy raises concern and hope

Immune system plays important role in Duchenne muscular dystrophy, research reveals

Immune system plays important role in Duchenne muscular dystrophy, research reveals

Sarepta Therapeutics wins accelerated approval from FDA for Duchenne muscular dystrophy drug

Sarepta Therapeutics wins accelerated approval from FDA for Duchenne muscular dystrophy drug

Researchers explore how ALS develops from muscle perspective

Researchers explore how ALS develops from muscle perspective

Research sheds light on how subtle genetic differences in DMD patients produce variation in symptoms

Research sheds light on how subtle genetic differences in DMD patients produce variation in symptoms

Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice

Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice

Researchers investigate biochemical, physiological characteristics of facial and extraocular muscles

Researchers investigate biochemical, physiological characteristics of facial and extraocular muscles

New approach could be used to treat Duchenne muscular dystrophy

New approach could be used to treat Duchenne muscular dystrophy

Duchenne muscular dystrophy: direct effect on muscle stem cells? An interview with Dr Rudnicki

Duchenne muscular dystrophy: direct effect on muscle stem cells? An interview with Dr Rudnicki