Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models

Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models

We previously showed correction of sickle cell anemia (SCA) in mice utilizing a lentiviral vector (LV) expressing human γ-globin. Herein, we made a G16D mutation in the γ-globin gene to generate the G16D mutation (GbGM) LV to increase fetal hemoglobin formation. We also generated an insulated versio...

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Bibliographic Details
Main Authors: Archana Shrestha, Devin M. Pillis, Sydney Felker, Mengna Chi, Kimberly Wagner, Oluwabukola T. Gbotosho, Joseph Sieling, Mohammad Shadid, Punam Malik
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
sickle cell anemia
lentiviral vector
human gamma-globin
fetal hemoglobin
insulator
hematopoietic stem cell
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125000348
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