Article

Using human genetics to develop strategies to increase erythropoietic output from genome-edited hematopoietic stem and progenitor cells

2023-08-04

Abstract excerpt

Human genetic polymorphisms result in a diversity of phenotypes. Some sequences are pathologic and lead to monogenic diseases, while others may confer beneficial traits. Genome editing is a powerful tool to recreate genotypes found in the population, including the ability to correct pathologic mutations. One of the best characterized naturally occurring mutations causing congenital erythrocytosis arises from a tru...

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Literature Corpus work: f58cf35c-1079-59ed-8b96-1ce6c951a880
DOI: 10.1101/2023.08.04.552064

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