Lab-Grown gene therapy shows promise for rare brain diseases
NCT ID NCT07445490
First seen Mar 07, 2026 · Last updated Apr 29, 2026 · Updated 10 times
Summary
This early-stage study tests a new gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases. Researchers will take blood cells from 6 patients, modify them in the lab, and see if they can help fix nerve cells grown from patient stem cells. The goal is to see if this approach could work as a treatment, but it is not yet a cure.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Département de Neurologie
Paris, 75013, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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