MTM1 and X-linked myotubular myopathy: In analogy, an AAV-mediated gene therapy for X-linked CNM (XLCNM) was first proven to be efficient on Mtm1 knockout mice (Buj-Bello et al., 2008) and validated on a spontaneous canine XLCNM model (Childers et al., 2014) prior to its usage in clinical trials (NCT03199469).