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gene therapy

The Bund skyline in Shanghai, where the medical school investigation was announced

Shanghai Medical School Investigates Undisclosed Death in Gene-Therapy Trial

A Shanghai medical school is investigating an undisclosed patient death tied in reports to an experimental gene-therapy procedure.

Huda Zoghbi, Director of the Jan and Dan Duncan Neurological Research Institute

Huda Zoghbi Opened a New Route to Rett Treatment. The Dose-Control Problem Will Decide Its Value

Huda Zoghbi’s latest Rett research attacks the disease’s narrow therapeutic window. Translating it will test whether precision neuroscience can deliver control, not simply a powerful one-time intervention.