AAV9-GAN (autonomic nervous system route)

AAV9-GAN (autonomic nervous system route) is a preclinical gene therapy candidate developed by the Hannah's Hope Fund (GAN Patient Advocacy Group) for the treatment of Giant Axonal Neuropathy (GAN). The therapy utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the GAN gene, which encodes the gigaxonin protein. This specific program is designed to target the autonomic nervous system to address critical, life-threatening symptoms of GAN, including impaired speech, swallowing difficulties, and involuntary breathing dysfunction. By restoring gigaxonin expression, the therapy aims to facilitate the proper degradation of intermediate filaments, preventing the axonal swelling and degeneration characteristic of the disease.

Other names

AAV9-GAN gene therapyAAV-9-GAN gene therapyAAV 9-GAN gene therapyHannah's Hope Fund GAN gene therapy