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Dysferlin (DYSF)

Target: DYSF
Molecular classification: Type II transmembrane protein, Ferlin family protein

Overview

Dysferlin is a large, type II transmembrane protein primarily expressed in skeletal muscle. It plays a critical role in membrane repair, especially of the sarcolemma, and is involved in vesicle trafficking, calcium homeostasis, and other cellular processes related to muscle function. Mutations in the DYSF gene cause dysferlinopathies, notably limb-girdle muscular dystrophy type 2B (LGMD2B) and Miyoshi myopathy, characterized by defective membrane repair and muscle degeneration.

Other names: FER1L1DYSF protein

Biological functions

Membrane repairVesicle traffickingVesicle fusionCalcium homeostasisAngiogenesis regulation

Disease associations

Limb-girdle muscular dystrophy type 2B (LGMD2B)Miyoshi myopathyDysferlinopathy

Safety considerations

Limited understanding of all dysferlin interactions, potentially leading to off-target effects.Challenges in delivering therapies to muscle tissue effectively.

Biomarkers

Dysferlin protein levels in muscle biopsiesGenetic mutations in DYSF gene

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