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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Detalles Bibliográficos
Autores principales: Iancu, Ortal, Allen, Daniel, Knop, Orli, Zehavi, Yonathan, Breier, Dor, Arbiv, Adaya, Lev, Atar, Lee, Yu Nee, Beider, Katia, Nagler, Arnon, Somech, Raz, Hendel, Ayal
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9813580/
https://www.ncbi.nlm.nih.gov/pubmed/36618262
http://dx.doi.org/10.1016/j.omtn.2022.12.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9813580/
https://www.ncbi.nlm.nih.gov/pubmed/36618262
http://dx.doi.org/10.1016/j.omtn.2022.12.006

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