The dysferlinopathies comprise a group of untreatable muscle disorders including limb
girdle muscular dystrophy type 2B, Miyoshi myopathy, distal anterior compartment …

Deficiency of the dysferlin protein presents as two major clinical phenotypes: limb–girdle
muscular dystrophy type 2B and Miyoshi myopathy. Dysferlin is known to participate in …

Recombinant adeno-associated virus (rAAV) is currently the best vector for gene delivery
into the skeletal muscle. However, the 5-kb packaging size of this virus is a major obstacle …

Objective Dysferlinopathies are a family of untreatable muscle disorders caused by
mutations in the dysferlin gene. Lack of dysferlin protein results in progressive dystrophy …

Dysferlinopathies are autosomal recessive, progressive muscle dystrophies caused by
mutations in DYSF, leading to a loss or a severe reduction of dysferlin, a key protein in …

Dysferlinopathies comprise a family of disorders caused by mutations in the dysferlin (DYSF)
gene, leading to a progressive dystrophy characterized by chronic muscle fiber loss, fat …

Dysferlin is a large transmembrane protein composed of a C-terminal transmembrane
domain, two DysF domains, and seven C2 domains that mediate lipid-and protein-binding …

Mutations in DYSF encoding dysferlin cause primary dysferlinopathies, autosomal recessive
diseases that mainly present clinically as Limb Girdle Muscular Dystrophy type 2B and …

Dysferlinopathy is an autosomal recessive muscular dystrophy characterized by the
progressive loss of motility that is caused by mutations throughout the DYSF gene. There are …

A drawback of gene therapy using adeno-associated virus (AAV) is the DNA packaging
restriction of the viral capsid (< 4.7 kb). Recent observations demonstrate oversized AAV …