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Adeno Associated virus serotype 8 (AAV8) is of particular interest as a vector for pre-clinical and clinical trial for Duchenne Muscular Dystrophy (DMD). In several cell lines, this vector has been shown to enter cells through clathrin-mediated endocytosis followed by a trafficking through the microtubule network in various endosomal compartments toward the nucleus. To efficiently transduce cells,...
The autosomal dominant centronuclear myopathy (AD-CNM) is a rare congenital myopathy defined by skeletal muscle weakness and characteristic histopathological changes. Heterozygous mutations in the DNM2 gene are associated with entire clinical spectrum of AD-CNM. DNM2 gene encodes dynamin 2 (DNM2), a large GTPase ubiquitously expressed and involved in membrane trafficking. A Knock-In mouse model (KI-Dnm2R465W)...