Article

TRACKING HEMATOPOIETIC STEM CELL EVOLUTION IN A WISKOTT-ALDRICH CLINICAL TRIAL

2022-05-31

Abstract excerpt

Hematopoietic Stem Cells (HSC) are the cells that give rise to all other blood cells and, as such, they are crucial in the healthy development of individuals. Wiskott-Aldrich Syndrome (WAS) is a severe disorder affecting the regulation of hematopoietic cells and is caused by mutations in the WASP gene. We consider data from a revolutionary gene therapy clinical trial, where HSC harvested from 3 WAS patients’ bone...

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Literature Corpus work: ad892a48-ee5d-595e-a59e-ab92f81a45e4
DOI: 10.1101/2022.05.30.494052

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