2022 Safadi Lecture: Emerging Gene Therapies for the Central Nervous System

Adeno-associated viruses (AAVs) have been given to many patients with inherited diseases, with few drugs achieving regulatory approval because of issues with durability and toxicity. These issues generally arise because AAVs applied clinically show poor gene delivery efficiency to target tissues and cells of interest, requiring high doses. Also, gene expression cannot be regulated once introduced to cells. This session will discuss ongoing research that demonstrates progress in addressing these critical issues for the broader utility of AAVs for human therapies. Learn more at our website!

After this activity, participants will be able to:

• Describe current gene therapy tools to treat brain diseases;
• Identify gain-of-function diseases, which are caused by the ectopic or increased activity of the mutated gene product;
• Recall recent improvements in gene vectors and their design that will result in improved outcomes for patients with brain diseases.