Scientists track 7 patients for 5 years after groundbreaking gene therapy
Disease control
Ongoing
This study monitors 7 patients for five years after they received an experimental gene therapy for Tay-Sachs or Sandhoff disease. The goal is to check for any long-term safety issues, like cancer or immune system problems, that might develop after treatment. Researchers will also…
Sponsor: Terence Flotte • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC