Beta-1,4-N-acetylgalactosaminyltransferase 2 (GALGT2)

Beta-1,4-N-acetylgalactosaminyltransferase 2 (GALGT2, also known as B4GALNT2) is an enzyme that catalyzes the transfer of N-acetylgalactosamine in a β1,4 linkage, specifically glycosylating α-dystroglycan in skeletal muscle. Overexpression of GALGT2, often achieved through AAV-mediated gene therapy, drives the glycosylation of this key membrane protein and induces the secondary upregulation of other membrane-stabilizing proteins, such as surrogate forms of dystrophin and laminin α2. This approach is being developed as a therapeutic strategy for certain neuromuscular diseases, including Duchenne muscular dystrophy and several limb-girdle muscular dystrophies, where increased membrane stabilization of muscle fibers is desirable. GALGT2-based therapies aim to reduce muscle degeneration and improve muscle function by fortifying the sarcolemma against mechanical damage. The therapeutic application of GALGT2 is subject to safety considerations such as off-target effects, species differences in glycan substrates, and potential immune responses to gene therapy vectors[1][3][7].

Other names

B4GALNT2GALGT2beta1,4-N-acetylgalactosaminyltransferase 2

Induces glycosylation of α-dystroglycan; upregulation leads to increased expression of membrane-stabilizing proteins that can compensate for deficient dystrophin or related proteins in muscular dystrophies[1][3][7]