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Results for “BCL11A”

6 trials

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Showing 6 of 6 results

Early research (Phase 1)Ended earlyNCT04443907
What this trial is testing

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

Who this might be right for
Sickle Cell Disease
Novartis Pharmaceuticals 4
Early research (Phase 1)Active Not RecruitingNCT06155500
What this trial is testing

Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

Who this might be right for
Sickle Cell Disease
Novartis Pharmaceuticals 4
Not applicableLooking for participantsNCT01238250
What this trial is testing

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

Who this might be right for
16P11.2 Deletion Syndrome16p11.2 Duplications1Q21.1 Deletion+181 more
Simons Searchlight 100,000
Early research (Phase 1)Looking for participantsNCT06647979
What this trial is testing

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

Who this might be right for
Sickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)Beta-Thalassemia+1 more
Daniel Bauer 10
Early research (Phase 1)Active Not RecruitingNCT03282656
What this trial is testing

Gene Transfer for Sickle Cell Disease

Who this might be right for
Sickle Cell Disease
David Williams 10
Testing effectiveness (Phase 2)Active Not RecruitingNCT05353647
What this trial is testing

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

Who this might be right for
Sickle Cell Disease
David Williams 25