In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies

Individuals with β-thalassemia or sickle cell disease and hereditary persistence of fetal hemoglobin (HPFH) possessing 30% fetal hemoglobin (HbF) appear to be symptom free. Here, we used a nonintegrating HDAd5/35++ vector expressing a highly efficient and accurate version of an adenine base editor (...

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Detalles Bibliográficos
Autores principales: Li, Chang, Georgakopoulou, Aphrodite, Newby, Gregory A., Everette, Kelcee A., Nizamis, Evangelos, 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: American Society for Clinical Investigation 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9675455/
https://www.ncbi.nlm.nih.gov/pubmed/36006707
http://dx.doi.org/10.1172/jci.insight.162939

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