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

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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Bibliographic Details
Main Authors: Chang Li, Aphrodite Georgakopoulou, Gregory A. Newby, Kelcee A. Everette, Evangelos Nizamis, Kiriaki Paschoudi, Efthymia Vlachaki, Sucheol Gil, Anna K. Anderson, Theodore Koob, Lishan Huang, Hongjie Wang, Hans-Peter Kiem, David R. Liu, Evangelia Yannaki, André Lieber
Format: Article
Language:English
Published: American Society for Clinical investigation 2022-10-01
Series:JCI Insight
Subjects:
Hematology
Stem cells
Online Access:https://doi.org/10.1172/jci.insight.162939

Internet

https://doi.org/10.1172/jci.insight.162939

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