CRISPR gene therapy offers hope for blood disorder patients
NCT ID NCT05477563
First seen Nov 06, 2025 · Last updated Apr 30, 2026 · Updated 29 times
Summary
This study tests a single treatment using CRISPR to modify a person's own blood stem cells, aiming to help them produce healthy red blood cells and reduce or eliminate the need for blood transfusions. It is for adults with transfusion-dependent beta-thalassemia or severe sickle cell disease. The approach involves collecting stem cells, editing them in a lab, and infusing them back after a short course of chemotherapy.
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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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IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
RECRUITINGRome, Italy
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King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology
RECRUITINGAl Mathar Ash Shamali, Saudi Arabia
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Levine Children's Hospital - Hematology
RECRUITINGCharlotte, North Carolina, 28203, United States
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New York Presbyterian Hospital - Morgan Stanley Children's Hospital
RECRUITINGNew York, New York, 10032, United States
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TriStar Medical Group Children's Specialists - Pediatric Oncology
RECRUITINGNashville, Tennessee, 37203, United States
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University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
RECRUITINGDüsseldorf, Germany
Conditions
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