AAV-CLN8

AAV-CLN8 is an investigational **AAV9-based in vivo gene therapy** designed to deliver a functional human **CLN8** transgene to the central nervous system for treatment of **CLN8 disease**, a form of neuronal ceroid lipofuscinosis also known as Batten disease. Preclinical reports describe a **self-complementary AAV9 vector** expressing human CLN8, administered directly into the cerebrospinal fluid and intended to restore CLN8 protein function in affected neurons and other CNS cells. The therapeutic rationale is gene replacement: pathogenic CLN8 loss impairs endoplasmic reticulum to Golgi trafficking of lysosomal enzymes, and replacement of CLN8 is intended to correct this upstream defect and reduce lysosomal storage pathology. The program appears to have originated at **Nationwide Children's Hospital** and is best documented in preclinical rather than established clinical development.

Other names

CLN8 gene therapyCLN-8 gene therapyCLN 8 gene therapyAAV9-CLN8AAV-9-CLN8AAV 9-CLN8scAAV9-CLN8scAAV-9-CLN8scAAV 9-CLN8