aavrh.10cuarsa

AAVrh.10cuARSA is an investigational gene therapy developed by Inserm (France) for the treatment of metachromatic leukodystrophy (MLD). It consists of an adeno-associated virus serotype rh.10 (AAVrh.10) vector that carries the human arylsulfatase A (ARSA) cDNA. Expression of the enzyme is driven by a cytomegalovirus/β-actin hybrid (CAG/cu) promoter. MLD is a fatal pediatric lysosomal storage disease caused by a deficiency in the ARSA enzyme, which leads to the toxic accumulation of sulfatides in the central nervous system and progressive demyelination. AAVrh.10cuARSA is administered via direct intracranial (intraparenchymal) injection into the white matter of both brain hemispheres. The therapy aims to restore enzyme activity, facilitate the breakdown of sulfatides, and prevent neurodegeneration through neuronal and oligodendrocyte transduction and subsequent axonal transport of the functional enzyme.

Other names

AAVrh.10-ARSAAAVrh10-ARSAAAVrh-10-ARSAAAVrh 10-ARSA