New Hope: Using Gene Therapy to Treat Rare Neurological Diseases

Abstract

Gene therapy is a promising treatment approach for rare/orphan neurological diseases that have limited treatment options and no cure. This article provides a brief overview of different types of rare hereditary neurological diseases and discusses existing gene therapy drugs approved for their treatment. Despite challenges associated with the development and implementation of gene therapy, including cost, delivery, and long-term safety and efficacy, the potential benefits of gene therapy make it a compelling area of research for the treatment of rare hereditary neurological diseases.