Article

Multiplex HDR for Disease and Correction Modeling of SCID by CRISPR Genome Editing in Human HSPCs

2022-10-07

Abstract excerpt

Severe combined immunodeficiency (SCID) is a group of monogenic primary immunodeficiencies caused by mutations in genes involved in the process of lymphocyte maturation and function. CRISPR-Cas9 gene editing of the patient’s own hematopoietic stem and progenitor cells (HSPCs) ex vivo could provide a therapeutic alternative to allogeneic hematopoietic stem cell transplantation (HSCT), the current gold standard for...

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Literature Corpus work: 18146777-d783-56e6-95a4-b954438d4c8b
DOI: 10.1101/2022.10.07.511272

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