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Optimized cell culture conditions promote <i>ex-vivo</i> manipulation and expansion of primitive hematopoietic stem cells for therapeutic gene editing

2022-01-11

Abstract excerpt

During the last few years, gene editing has emerged as a powerful tool for the therapeutic correction of monogenic diseases. CRISPR/Cas9 applied to hematopoietic stem and progenitor cells (HSPCs) has shown great promise in proof-of-principle preclinical studies to treat haematological disorders, and clinical trials using these tools are now underway. Nonetheless, there remain important challenges that need to be a...

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Literature Corpus work
b66ce3e1-0d2e-50f1-b9b8-fa0d4695d98a
DOI
10.1101/2022.01.11.475795
Open publication

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Optimized cell culture conditions promote <i>ex-vivo</i> manipulation and expansion of primitive hematopoietic stem cells for therapeutic gene editingDOI 10.1101/2022.01.11.475795
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