Volume 27 Issue 10-11, November 2020

Desminopathies are a therapeutic challenge as there are over 130 different mutations in the human desmin gene resulting in myopathy and/or cardiomyopathy. In this issue, a gene therapeutic approach is validated in two different models of autosomal-recessive desminopathy. This image shows successful AAV-mediated reconstitution of myocardial desmin filaments in desmin deficient mice (left to right: wildtype mice; desmin deficient control mice; AAV-treated desmin deficient mice). See paper by Ruppert et al. in this issue of Gene Therapy.