Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood Disorders

Genome engineering via targeted nucleases, specifically CRISPR-Cas9, has revolutionized the field of gene therapy research, providing a potential treatment for diseases of the blood and immune system. While numerous genome editing techniques have been used, CRISPR-Cas9 homology-directed repair (HDR)...

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Detalles Bibliográficos
Autores principales: Allen, Daniel, Kalter, Nechama, Rosenberg, Michael, Hendel, Ayal
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10220672/
https://www.ncbi.nlm.nih.gov/pubmed/37242571
http://dx.doi.org/10.3390/pharmaceutics15051329

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10220672/
https://www.ncbi.nlm.nih.gov/pubmed/37242571
http://dx.doi.org/10.3390/pharmaceutics15051329

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