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

• Main Authors: Ortal Iancu (Author), Daniel Allen (Author), Orli Knop (Author), Yonathan Zehavi (Author), Dor Breier (Author), Adaya Arbiv (Author), Atar Lev (Author), Yu Nee Lee (Author), Katia Beider (Author), Arnon Nagler (Author), Raz Somech (Author), Ayal Hendel (Author)
• Format: Book
• Published: Elsevier, 2023-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 alternati...