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    CRISPR Halts Duchenne Muscular Dystrophy Progression in Dogs

    September 1, 2018, Categories  Biology/Biotechnology, Health/Medical

    Scientists used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in dogs. The images illustrate dystrophin (in green) in a healthy diaphragm muscle (left), absence of dystrophin in a dog with DMD (center), and restoration of dystrophin in dogs treated with CRISPR (right). Credit: UT Southwestern

    Scientists used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in dogs. The images illustrate dystrophin (in green) in a healthy diaphragm muscle (left), absence of dystrophin in a dog with DMD (center), and restoration of dystrophin in dogs treated with CRISPR (right).
    Credit: UT Southwestern

    Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline...

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    Gene Therapy treats all Muscles in the Body in Muscular Dystrophy Dogs

    October 22, 2015, Categories  Biology/Biotechnology, Health/Medical

    The dystrophin protein connects the cytoskeleton of muscle fibers with its N-terminal actin-binding domain and the extra-cellular matrix via a cysteine-rich C-terminal domain that binds dystroglycan, which in turn is bound to laminin 2. Between these two functional domain lies a large central domain that consists of 24 spectrin like repeats.

    The dystrophin protein connects the cytoskeleton of muscle fibers with its N-terminal actin-binding domain and the extra-cellular matrix via a cysteine-rich C-terminal domain that binds dystroglycan, which in turn is bound to laminin 2. Between these two functional domain lies a large central domain that consists of 24 spectrin like repeats.

    Human clinical trials are next step. Muscular dystrophy, which affects approximately 250,000 people in the US, occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy (DMD), which primarily affects boys...

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