One-Shot gene editing could end debilitating pain crises

NCT ID NCT05456880

Summary

This study is testing a new, one-time treatment for severe sickle cell disease. Doctors take a patient's own blood stem cells, edit their genes in a lab, and then return them to the patient. The goal is to see if this single treatment can safely and significantly reduce the number of painful and dangerous blood vessel blockages (crises) that patients experience.

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

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02215, United States

  • Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center - Egleston Hospital

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Henry Ford Cancer Center

    Detroit, Michigan, 48202, United States

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224, United States

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • St Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • The Children's Hospital at TriStar Centennial

    Nashville, Tennessee, 37203, United States

  • The Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106, United States

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233, United States

  • University of Miami

    Miami, Florida, 33136-1005, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • Washington University School of Medicine in St. Louis

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.