PerspectiveMedicine

RNA-targeted drugs for neuromuscular diseases

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Science  01 Jan 2021:
Vol. 371, Issue 6524, pp. 29-31
DOI: 10.1126/science.aba4515

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Summary

Neuromuscular diseases (NMDs) are common and heterogeneous conditions that either affect skeletal muscle directly, as in muscular dystrophies, or affect motor neurons, peripheral nerves, or neuromuscular junctions. The abundance of skeletal muscle and the size of several of the genes that cause NMDs limit the application of adeno-associated virus–delivered gene therapy (1). Manipulation of RNA to correct mutated transcripts has been used successfully in several NMDs, leading to approved first-generation drugs; next-generation drugs are now in clinical development. But the development of RNA therapies has also been accompanied by several failures, highlighting problems of safety, efficacy, and tissue targeting that need to be overcome.

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