Article

Clinically Relevant Gene Editing in Hematopoietic Stem Cells for the Treatment of Pyruvate Kinase Deficiency Hemolytic Anemia

2021-01-15

Abstract excerpt

<h4>ABSTRACT</h4> Pyruvate Kinase Deficiency (PKD) is an autosomal recessive disorder caused by mutations in the PKLR gene, which constitutes the main cause of chronic non-spherocytic hemolytic anemia. PKD incidence is estimated in 1 in 20,000 people worldwide. The PKLR gene encodes for the erythroid pyruvate kinase protein (RPK) implicated in the last step of the anaerobic glycolysis in red blood cells. The de...

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Literature Corpus work: 8e7c4def-09eb-53ef-83b0-414b998f7a23
DOI: 10.1101/2021.01.14.426673

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