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Genome Editing Therapy for the Blood: Ex Vivo Success and In Vivo Prospects

26 Sept 2024

Abstract excerpt

Hematopoietic stem cells (HSCs) provide the body with a continuous supply of healthy, functional blood cells. In patients with hematopoietic malignancies, immunodeficiencies, lysosomal storage disorders, and hemoglobinopathies, therapeutic genome editing offers hope for corrective intervention, with even modest editing efficiencies likely to provide clinical benefit. Engineered white blood cells, such as T cells,...

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PMID: 39324895
DOI: 10.1089/crispr.2024.0036

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