Article

Correction of the Sickle Cell Mutation Through Base and Prime Editing in Hematopoietic Stem Cells

2020-09-21

Abstract excerpt

Sickle cell disease is characterized by stiff, “sickled” red blood cells that have difficulty moving through the bloodstream and do not efficiently carry oxygen. It is an inherited disease with severely limited treatment options, and is caused by a point mutation. Its prevalence in black and brown communities makes the already limited treatment options even less accessible. Base editing and prime editi...

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Literature Corpus work: ab33212f-2f0a-5008-906a-b982376c449e
DOI: 10.20944/preprints202009.0490.v1

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