ASGCT 2021 | Strategies for advancing gene therapy for CNS disorders

Kathrin Meyer, PhD, Nationwide Children’s Hospital, Columbus, OH, discusses ongoing research into enhancing gene therapies for central nervous system (CNS) disorders, such as through improvement of vector capsid efficiency to reduce the dosage required. Improved methods for therapeutic delivery are also being investigated including ultrasound-guided delivery systems, novel vector formulations and alternative transplantation sites, such as the cisterna magna and intraparenchymal injection. In addition, mathematical modelling and biophysics are being applied to enhance the systemic distribution of cerebrospinal fluid (CSF) delivered therapeutics to prevent hotspot formation and ensure complete coverage of the brain. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.