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Gene editing study reveals possible 'Achilles heel' of sickle cell disease


September 16, 2015

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Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, using CRISPR-based, gene-editing tools, have found that changes to a small stretch of DNA may circumvent the genetic defect behind sickle cell disease (SCD). The discovery, published in the journal Nature, creates a path for developing gene editing approaches for treating SCD and other hemoglobin disorders, such as thalassemia.Full Story

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Dana-Farber Cancer Institute

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