Molecular Therapy For Spinal Muscular Atrophy Closer To Clinical Use

Spinal muscular atrophy, a neurodegenerative disorder that causes the weakening of muscles, is the leading cause of infant death and occurs in 1 in 6,000 live births. While trans-splicing (a form of molecular therapy) has had impressive results as a treatment for spinal muscular atrophy in cell-based models of disease, scientists have been unable to translate the therapy to the human body. Researchers have now developed a strategy that will enhance trans-splicing activity and bring it closer to being used in the clinical setting.

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