Canavan Disease
This article highlights the possible usage of rAAV-Olig001-ASPA Gene Therapy created by Myrtelle for treating Canavan Disease (CD). Specifically, CD is an inherited condition caused by mutations in the Aspartoacylase gene (ASPA) which is responsible for producing the enzyme Aspartoacylase (ASPA) leading to abnormally low quantities of the enzyme in neural cells. That results in the accumulation of ASPA in neural cells and the effects can be devasting as the protective sheath covering neurons (known as myelin) is destroyed. This affects the ability of the neurons to communicate and transfer the information from the brain to organs and cells. As a result, CD can cause irreversible damage, and as there is no available treatment it can be life limiting. However, Myrtelle was able to develop a gene therapy that could treat CD. By the term gene therapy, scientists refer to the insertion of functional genes to restore their normal function. Patients that were administered the innovative treatment showed positive data indicating that it could restore the function of ASPA. Specifically, gene therapy for CD targets directly the neural cells allowing normal production of ASPA. Overall, a re-evaluation of the new gene therapy should be made to ensure the safety and efficacy of the drug before used to treat CD.