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CRISPR breakthrough trial offers hope for genetic blood disease cure

NCT ID NCT05477563

Recruiting now Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

Summary

This study is testing a single treatment using CRISPR gene editing on a patient's own blood stem cells to treat severe sickle cell disease or transfusion-dependent thalassemia. The goal is to help the body produce healthy hemoglobin and reduce or eliminate the need for blood transfusions and painful crises. Researchers will follow 26 participants to see if this one-time treatment is safe and effective long-term.

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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica

    RECRUITING

    Rome, Italy

  • King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology

    RECRUITING

    Al Mathar Ash Shamali, Saudi Arabia

  • Levine Children's Hospital - Hematology

    RECRUITING

    Charlotte, North Carolina, 28203, United States

  • New York Presbyterian Hospital - Morgan Stanley Children's Hospital

    RECRUITING

    New York, New York, 10032, United States

  • TriStar Medical Group Children's Specialists - Pediatric Oncology

    RECRUITING

    Nashville, Tennessee, 37203, United States

  • University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology

    RECRUITING

    Düsseldorf, Germany

Conditions

Explore the condition pages connected to this study.

BETA-THALASSEMIA GENETIC DISEASES, INBORN HEMATOLOGIC DISEASES HEMOGLOBINOPATHIES SICKLE CELL ANEMIA SICKLE CELL DISEASE Β-THALASSEMIA