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Investigational curative gene therapy approaches to sickle cell disease

14 Dec 2021

Abstract excerpt

Sickle cell disease (SCD) is an inherited blood condition resulting from abnormal hemoglobin production. It is one of the most common genetic diseases in the world. The clinical manifestations are variable and range from recurrent acute and debilitating painful crises to life-threatening pulmonary, cardiovascular, renal, and neurologic complications. The only curative treatment of SCD at this time is bone marrow...

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PMID: 34905048
DOI: 10.1182/bloodadvances.2021005567

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