Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle

KE Wells, S Torelli, Q Lu, SC Brown, T Partridge… - Neuromuscular …, 2003 - Elsevier
A lack of effective treatments for Duchenne muscular dystrophy, a fatal X-linked myopathy,
has focused attention on the possibility of gene therapy. The aim of the gene therapy
approach is the restoration of the dystrophin associated complex of proteins, one member of
which is neuronal nitric oxide synthase, an important enzyme in signal transduction.
Transgenic mdx mice and plasmid gene transfer of both human and murine recombinant
dystrophins was used to assess whether nNOS could be restored to the sarcolemma …