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3 years ago

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CRISPR Gene Editing Delivers Promise for Sickle-Cell Disease, Beta Thalassemia

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CRISPR Gene Editing Delivers Promise for Sickle-Cell Disease, Beta Thalassemia

CRISPR Therapeutics and Vertex Pharmaceuticals have reported a consistent and sustained positive response in ten patients treated for a pair of blood disorders—sickle-cell disease (SCD) and beta thalassemia—with their CRISPR-Cas9 gene-edited therapy CTX001 in a pair of Phase I/II trials. These are the first clinical studies of a CRISPR gene-editing candidate sponsored by U.S. companies.

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CRISPR Gene Editing Delivers Promise for Sickle-Cell Disease, Beta Thalassemia

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CRISPR Gene Editing Delivers Promise for Sickle-Cell Disease, Beta Thalassemia

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