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A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

Vertex Pharmaceuticals Incorporated · 2018
ClinicalTrials.gov · Datasets · License: Public Domain · 2018
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crispr-cas9genetic-diseases
beta-thalassemia, thalassemia, genetic diseases, inborn, hematologic diseases, hemoglobinopathies, crispr-cas9, interventional, phase2, phase3
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