Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of ...
Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new ...
Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. Studies of the new protein ...
Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...
People with Duchenne muscular dystrophy carry a mutation in the DMD gene coding for dystrophin, a protein crucial for proper muscle function. Scientists are working to treat the disease at its genetic ...
Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for ...
Duchenne muscular dystrophy (DMD) is a genetic disease that causes progressive muscle weakness. DMD commonly affects boys, where symptoms are seen in early childhood. There is no cure for DMD. Health ...
An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne muscular dystrophy, a deadly and irreversible disease, ...
Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of ...
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section ...
Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. Studies of the new protein ...
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