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CRISPR-Cas9 Gene Editing for Treatment of Hereditary Blood Disorders
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Keywords: CRISPR gene editing thalassemia sickle cell clinical trial
Abstract
We report successful application of CRISPR-Cas9 gene editing technology in treating beta-thalassemia and sickle cell disease in a Phase II clinical trial involving 48 patients. Results show 87% of patients achieved transfusion independence at 12-month follow-up with manageable off-target effects.
Authors & Affiliations
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Prof. Sarah Chen
(Corresponding)
Massachusetts Institute of Technology, USA
ORCID: 0000-0002-3456-7890
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