Scientists test modified blood cells as potential lifeline for devastating brain diseases
NCT ID NCT07445490
Summary
This early-stage study is testing a new gene therapy method for Tay-Sachs and Sandhoff disease, two rare and fatal neurological disorders. Researchers will modify patients' blood stem cells in a lab to produce a missing enzyme, then test if these cells can help correct brain cells in lab dishes. The goal is to develop a treatment that could slow or stop disease progression for conditions that currently have no cure.
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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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