Gene Therapy
Ethical issues surrounding gene therapy trials and proposed clinical trial endpoints for gene therapy for NF1, NF2, and Schwannomatosis.
Leadership
About This 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
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`
Additional Information
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Working Group Snapshot & Focus Updates
The Gene Therapy group was formed in January 2022. Initial projects focus on defining the important ethical issues surrounding gene therapy in the NF/SWN patient population and establishing appropriate, measurable endpoints for future gene therapy clinical trials.
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Documents & Resources
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Join or Contact This Group
Membership is open to all interested parties. Meetings are primarily held by teleconference.
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