One-Time gene edit aims to cure devastating blood diseases
NCT ID NCT06647979
Summary
This early-stage trial is testing a one-time gene editing treatment for people with severe sickle cell disease or beta-thalassemia. Doctors take a patient's own blood stem cells, edit a specific gene to boost healthy fetal hemoglobin, and return the cells after chemotherapy. The goal is to create a lasting cure by fixing the genetic cause of these painful and life-limiting conditions.
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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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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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