Gene Therapy Group

The goals of the gene therapy working group are (1) to discuss ethical issues surrounding trials of gene therapy and (2) to propose clinical trial endpoints for gene therapy that can lead to drug approval for NF1, NF2, and SWN.

Current group leader: Scott Plotkin, MD, PhD

Current Work

The Gene Therapy group was formed in January 2022 and will start by defining goals for the year.  Initial projects will focus on defining the important ethical issues surrounding gene therapy in the NF/SWN patient population.

last updated by Lessing, A on Dec 12, 2025