Stanley C. FROEHNER,Marvin ADAMS,Min Jeong KIM,Daniela Rebolledo LOPEZ,Nicholas P. WHITEHEAD
申请号:
US14916429
公开号:
US20160220647A1
申请日:
2014.09.09
申请国别(地区):
US
年份:
2016
代理人:
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.