The Silverman lab was well-represented at the annual FAST Summit in 2019.

A Therapeutic approach to treating Angelman syndrome using hematopoietic stem cell (HSC) gene replacement therapy.

A novel approach to gene replacement therapy through the use of genetically modified hematopoietic stem cells (HSCs) that have the potential to deliver functional UBE3A protein to affected cells throughout the brain. This presentation will focus on both preclinical data and the clinical approach that will be taken to evaluate this potential therapeutic.