Article

Efficient Correction of the Sickle Mutation in Human Hematopoietic Stem Cells Using a Cas9 Ribonucleoprotein Complex

2016-01-15

Abstract excerpt

Sickle Cell Disease (SCD) is a serious recessive genetic disorder caused by a single nucleotide polymorphism (SNP) in the ß-globin gene ( HBB ). Sickle hemoglobin polymerizes within red blood cells (RBCs), causing them to adopt an elongated “sickle” shape. Sickle RBCs damage vasculature, leading to severe symptoms, ultimately diminishing patient quality of life and reducing lifespan. Here, we use codelivery of a...

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Literature Corpus work: c1f4b44a-a164-5218-8cec-dc51a587edaf
DOI: 10.1101/036236

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