Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

Calpains are a family of proteases that were scientifically recognized earlier than
proteasomes and caspases, but remain enigmatic. However, they are known to participate in …

Proteases are an expanding class of drugs that hold great promise. The US FDA (Food and
Drug Administration) has approved 12 protease therapies, and a number of next generation …

Pediatric neuromuscular diseases encompass all disorders with onset in childhood and
where the primary area of pathology is in the peripheral nervous system. These conditions …

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 …

Calpains are Ca 2+-regulated proteolytic enzymes that are involved in a variety of biological
phenomena. Calpains process substrates by limited proteolysis to modulate various protein …

Calpains are a family of Ca 2+‑dependent cysteine proteases that participate in various
cellular processes. Calpain 3 (CAPN3) is a classical calpain with unique N‑terminus and …

Calpain 3 is a 94‐kDa calcium‐dependent cysteine protease mainly expressed in skeletal
muscle. In this tissue, it localizes at several regions of the sarcomere through binding to the …

There has been an ever-expanding list of the Limb–Girdle Muscular Dystrophies (LGMD).
There are currently 8 subtypes of autosomal dominant (AD) and 26 subtypes of autosomal …

Limb-girdle muscular dystrophies (LGMDs) represent a major group of muscle disorders.
Treatment is sorely needed and currently expanding based on safety and efficacy adopting …