Medical research

Duchenne muscular dystrophy is a stem cell disease

A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments.

Genetics

CRISPR enhances gene therapy to fight inherited diseases

Gene therapy has shown promise in treating inherited genetic diseases, but a major issue that has frustrated scientists remains: Replacing a "bad" gene with a healthy one often is a short-lived fix. Typically, the healthy ...

Genetics

Team identifies gene mutations behind lack of a nose

Researchers from Massachusetts General Hospital (MGH) led a large, international research team that has identified gene mutations associated with a rare congenital condition involving the absence of a nose and often accompanied ...

Genetics

Mutation found in patients without a nose

A mutated gene in patients lacking a nose has been identified by an international team, a first step toward understanding nose development and possible therapies for another condition.

Medical research

New muscular dystrophy treatment shows promise in early study

A preclinical study led by researchers in the United States has found that a new oral drug shows early promise for the treatment of muscular dystrophy. The results, which are published today in EMBO Molecular Medicine, show ...

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