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.
Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Single CRISPR treatment can safely and stably correct genetic disease

Single CRISPR treatment can safely and stably correct genetic disease

Study findings hold promise for children with DMD

Study findings hold promise for children with DMD

Researchers identify and overcome barrier in CRISPR gene editing to treat muscular dystrophy

Researchers identify and overcome barrier in CRISPR gene editing to treat muscular dystrophy

Urine exRNA may be source of biomarkers for muscular dystrophy

Urine exRNA may be source of biomarkers for muscular dystrophy

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Muscle dystrophy treatment in dogs with Crispr gene editing

Muscle dystrophy treatment in dogs with Crispr gene editing

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

New project to combat DMD-related fibrosis receives major funding boost

New project to combat DMD-related fibrosis receives major funding boost

New cell-based technologies could help improve understanding of muscle-wasting disease

New cell-based technologies could help improve understanding of muscle-wasting disease

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy

New guidelines intend to provide template for optimal, most up-to-date DMD care

New guidelines intend to provide template for optimal, most up-to-date DMD care

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows

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