PROTEINS FOR TARGETING NEURONAL NITRIC OXIDE SYNTHASE TO MUSCLE SARCOLEMMA AND RELATED METHODS OF USE

摘要

It is demonstrated herein that nNOS-μ is an important modulator of muscle function, and that the loss of nNOS-μ contributes to pathogenesis in neuromuscular diseases, including DMD. Furthermore, the impact of the restoration of cytoplasmic and sarcolemma-localized nNOS-μ on mdx skeletal muscle pathology and function is demonstrated. Accordingly, provided herein are compositions, including therapeutic and pharmaceutical compositions, comprising nNOS-μ fusion proteins and derivatives thereof, and methods of treating neuromuscular disorders using such nNOS-μ fusion proteins.