MTM1 and X-linked myotubular myopathy: Outside the use of gene therapy for hematological diseases, the role of preexisting liver disease has also been emphasized in the context of a clinical trial evaluating AT132 (an AAV8 vector containing a functional copy of the MTM1 gene) in patients with X-linked myotubular myopathy (XLMTM) (Philippidis, 2020).