Innovations

Using RNA interference to correct a mistake in the genetic code of patients with Fukuyama muscular dystrophy

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Focused chemists in lab coats and safety goggles making experiments.

Muscular dystrophy is a debilitating disease that causes progressive weakening and loss of muscles. Fukuyama congenital muscular dystrophy (FCMD), the second most common form of childhood muscular dystrophy in Japan, is a severe neuromuscular disorder characterized by generalized muscle weakness, decreased muscle tone, eye abnormalities, brain malformation, cardiomyopathy, epilepsy, and seizures associated with intellectual disability.

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