Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs

Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs

Severe combined immunodeficiency (SCID) is a group of disorders 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...

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Bibliographic Details
Main Authors: Ortal Iancu, Daniel Allen, Orli Knop, Yonathan Zehavi, Dor Breier, Adaya Arbiv, Atar Lev, Yu Nee Lee, Katia Beider, Arnon Nagler, Raz Somech, Ayal Hendel
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
CRISPR-Cas9
rAAV6
HSPCs
genome editing
gene regulation
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122003237

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http://www.sciencedirect.com/science/article/pii/S2162253122003237

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