Adeno-associated virus (AAV) vectors are one of the most efficient in vivo gene delivery
platforms. Over the past decade, clinical trials of AAV vector-mediated gene transfer led to …

Limb-girdle muscular dystrophies (LGMD) are a heterogeneous group of disorders, which
has led to certain investigators disputing its rationality. The mutual feature of LGMD is limb …

Loss-of-function mutations in the myotubularin gene (MTM1) cause X-linked myotubular
myopathy (XLMTM), a fatal, congenital pediatric disease that affects the entire skeletal …

Duchenne muscular dystrophy is a monogenic disease potentially treatable by gene
replacement. Use of recombinant adeno-associated virus (AAV) will ultimately require a …

Treating muscle disorders poses several challenges to the rapidly evolving field of
regenerative medicine. Considerable progress has been made in isolating, characterizing …

Gene therapy for the muscular dystrophies has evolved as a promising treatment for this
progressive group of disorders. Although corticosteroids and/or supportive treatments …

Over‐expression of the dystrophin‐related gene utrophin represents a promising therapeutic
strategy for Duchenne muscular dystrophy (DMD). The strategy is based on the ability of …

The development of clinical vectors to correct genetic mutations that cause inherited
myopathies and related disorders of skeletal muscle is advancing at an impressive rate …

Preventing untoward immune responses against a specific antigen is a major challenge in
different clinical settings such as gene therapy, transplantation, or autoimmunity. Following …

Генетические заболевания, приводящие к первичному поражению скелетной
мышечной ткани, могут быть обусловлены дисфункцией более чем 30 генов. Сегодня …