Arylsulfatase B mRNA (ARSB mRNA)

Arylsulfatase B (ARSB) mRNA is the messenger RNA transcript that encodes the lysosomal enzyme N-acetylgalactosamine-4-sulfatase (UniProt: P15848). This enzyme is critical for the catabolism of glycosaminoglycans (GAGs), specifically dermatan sulfate and chondroitin 4-sulfate (PubMed: 25578333). Deficiencies in the ARSB protein, caused by mutations in the ARSB gene, result in Mucopolysaccharidosis VI (MPS VI), also known as Maroteaux-Lamy syndrome, a progressive lysosomal storage disorder (NIH: Genetic and Rare Diseases Information Center). Therapeutic targeting of ARSB mRNA primarily involves mRNA replacement therapy, where exogenous, synthetic mRNA is delivered to the patient's cells to facilitate the production of functional ARSB enzyme. This approach aims to bypass the genetic defect and alleviate the systemic accumulation of GAGs that leads to skeletal deformities, cardiac issues, and respiratory failure (PubMed: 31216320). Clinical candidates like mRNA-3926 utilize lipid nanoparticle technology to deliver the ARSB mRNA, representing a novel alternative to traditional enzyme replacement therapies (Moderna: Pipeline).

Other names

Arylsulfatase B messenger RNAN-acetylgalactosamine-4-sulfatase mRNAASB mRNA

mRNA replacement therapy to restore functional Arylsulfatase B enzyme production