CNS Targeted Gene Therapies

The rapid advances in the field of gene therapy in the past decade have facilitated the rapid development of many treatments for previously untreatable neurological diseases.Gene therapy creates a long-term treatment for these diseases in comparison to other traditional medications.In recent years, numerous neurodegenerative diseases such as Parkinson's Disease and Alzheimer's Disease have been targets of gene therapy.Alongside the improved efficacy of gene therapy treatments, advancements in efficient vectors of delivery have proved instrumental to the field.This review provides an overview of gene therapy vectors and their delivery methods to the Central Nervous System (CNS).Additionally, this review rationalizes the necessity for further research into promising vectors, such as exosomes for CNS-targeted drugs.Finally, this review highlights the recently developed technology of ultrasound-mediated blood-brain barrier (BBB) disruption which has proven itself to be a promising alternative to traditionally developed CNS targeting mechanisms.