Canavan Disease
https://clinicaltrials.gov/ct2/show/NCT04833907
The purpose of this study is to validate a new technology targeted to the cells most affected by Canavan Disease in the safest way possible. Canavan Disease is a congenital white matter disorder caused by mutations to the gene encoding for aspartoacylase (ASPA) which expression is restricted to the oligodendrocytes. This protocol directly targets oligodendrocytes in the brain, which are intimately involved with disease initiation and progression. Targeting oligodendrocytes offers the safest and most direct therapy for affected individuals.
The latest generation AAV viral vector (rAAV-Olig001-ASPA) involves direct administration of gene therapy to affected regions of the brain. Patients with a diagnosis of typical Canavan Disease who meet all eligibility criteria may be enrolled.