Virus vector mediated gene therapy for the treatment of X-linked myotubular myopathy in dogs

Virus vector mediated gene therapy for the treatment of X-linked myotubular myopathy in dogs

A paper published earlier this year in Molecular Therapy detailed a successful dose dependent response of a usually fatal monogenic disorder of skeletal muscle in dogs to gene therapy. The disorder treated was X-Linked Myotubular Myopathy (XLMTM) which results from MTM1 gene mutations and deficiency in the protein that this gene encodes (myotubularin).