AAV-EIF2B5

AAV-EIF2B5 is an experimental, preclinical gene-supplementation therapy designed to treat vanishing white matter (VWM) disease, a fatal childhood leukodystrophy. VWM is caused by autosomal recessive mutations in the genes encoding the eukaryotic initiation factor 2B (eIF2B) complex, most commonly in the EIF2B5 subunit, which leads to astrocytic dysfunction and progressive demyelination. Developed by researchers at Nationwide Children's Hospital and the University of Utah, the therapy utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the human EIF2B5 gene. The lead construct, AAV9-gfaABC(1)D-EIF2B5, employs a truncated glial fibrillary acidic protein (GFAP) promoter to specifically target astrocytes. In preclinical mouse models of VWM, the therapy has demonstrated the ability to improve motor function, normalize gait, attenuate demyelination, and significantly extend survival.

Other names

AAV9-CAG-EIF2B5AAV-9-CAG-EIF2B5AAV 9-CAG-EIF2B5AAV9-GFAP-EIF2B5AAV-9-GFAP-EIF2B5AAV 9-GFAP-EIF2B5AAV9-gfa1405-EIF2B5AAV-9-gfa1405-EIF2B5AAV 9-gfa1405-EIF2B5AAV9-gfaABC(1)D-EIF2B5