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Fukutin-related protein (FKRP)
- Target
- FKRP
- Molecular classification
- Enzyme, Glycosyltransferase, Ribitol-5-phosphate transferase
01
Overview
Fukutin-related protein (FKRP) is a glycosyltransferase enzyme crucial for the functional glycosylation of alpha-dystroglycan (α-DG), a key protein that links the cytoskeleton inside cells to the extracellular matrix (ECM). Mutations in FKRP disrupt normal glycosylation of α-dystroglycan, leading to a group of disorders known as dystroglycanopathies. Potential therapeutic approaches being explored include small molecule drugs targeting defective pathways, gene therapy approaches delivering functional copies of *FKRP*, and cell-based therapies aiming to restore normal protein function or replace affected tissues.
- Other names
- LGMD2IMDC1CMDDGA5MDDGB5MDDGC5LGMDR9
02
Mechanism of action
N/A
03
Biological functions
Glycosylation of alpha-dystroglycanPost-translational modificationCell adhesionExtracellular matrix organizationNeuronal migration
04
Disease associations
Limb-girdle muscular dystrophyCongenital muscular dystrophyWalker-Warburg syndromeMuscle-eye-brain diseaseDystroglycanopathy
05
Safety considerations
Targeting glycosylation pathways may have broad effectsDelivery of functional FKRP to affected tissues is a challenge
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