One-Shot CRISPR treatment aims to free kids from sickle cell pain
NCT ID NCT05329649
Summary
This study is testing a single dose of an experimental gene therapy called CTX001 in children with severe sickle cell disease. The goal is to see if this one-time treatment can safely prevent the painful blockages in blood vessels (crises) that define the disease. The therapy works by using CRISPR gene-editing technology to modify a patient's own blood stem cells before putting them back into the body.
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Contacts and locations
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Locations
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IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
Rome, Italy
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Levine Children's Hospital - Hematology
Charlotte, North Carolina, 28203, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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St.Mary's Hospital - Haematology Dept
London, United Kingdom
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The Children's Hospital of Philadelphia - Hematology
Philadelphia, Pennsylvania, 19104, United States
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TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, 37203, United States
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University Hospital Duesseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
Düsseldorf, Germany
Conditions
Explore the condition pages connected to this study.