In vivo HSC prime editing rescues sickle cell disease in a mouse model

Sickle cell disease (SCD) is a monogenic disease caused by a nucleotide mutation in the β-globin gene. Current gene therapy studies are mainly focused on lentiviral vector–mediated gene addition or CRISPR/Cas9–mediated fetal globin reactivation, leaving the root cause unfixed. We developed a vectori...

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Detalles Bibliográficos
Autores principales: Li, Chang, Georgakopoulou, Aphrodite, Newby, Gregory A., Chen, Peter J., Everette, Kelcee A., Paschoudi, Kiriaki, Vlachaki, Efthymia, Gil, Sucheol, Anderson, Anna K., Koob, Theodore, Huang, Lishan, Wang, Hongjie, Kiem, Hans-Peter, Liu, David R., Yannaki, Evangelia, Lieber, André
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The American Society of Hematology 2023
Materias:
Gene Therapy
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10163316/
https://www.ncbi.nlm.nih.gov/pubmed/36800642
http://dx.doi.org/10.1182/blood.2022018252

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