Decades of biological research have unveiled the genetic cause of many debilitating diseases that affect the central nervous system (CNS). We are at a point where we aim to translate that knowledge into potential one-time treatment options and  curative therapies.  Gene therapy as a therapeutic modality is continuing to evolve and we have seen important advances propelling the field forward. However, additional focused innovation is needed to overcome the complex challenges in gene therapy for neurological diseases. Challenges regarding the delivery platform, cargo, vector production, and vector performance characterization as well as potential solutions to these challenges will be discussed.

Gene therapy for neurological diseases

ACS Fall 2021

The official archive and repository for all ACS Meetings & Expositions abstracts and select posters or presentations.