DOK7 gene therapy benefits mouse models of diseases characterized by defects in the neuromuscular junction

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Science  19 Sep 2014:
Vol. 345, Issue 6203, pp. 1505-1508
DOI: 10.1126/science.1250744

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Building connections by gene therapy

Voluntary movement requires a molecular conversation between nerves and muscles. This conversation occurs at the neuromuscular junction, a structure where nerves and muscle physically connect. People with diseases characterized by muscle weakness have aberrantly small neuromuscular junctions. Arimura et al. used gene therapy to enlarge the neuromuscular junction, which made muscles stronger. Studying mouse models of two distinct neuromuscular disorders, they used an adenovirus vector to deliver DOK7, a gene required for formation of the neuromuscular junction. The therapy improved the mice's motor activity and life span.

Science, this issue p. 1505