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Frontiers in Genome Editing|October 29, 2021
Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem CellsElizabeth K Benitez, Anastasia Lomova Kaufman, Lilibeth Cervantes, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 20, 2019
Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region ElementsRichard A Morgan, Mildred J Unti, Bamidele Aleshe, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 22, 2013
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiencyDenise A Carbonaro, Lin Zhang, Xiangyang Jin, et al.
The Journal of Clinical Investigation|July 19, 2013
β-globin gene transfer to human bone marrow for sickle cell diseaseZulema Romero, Fabrizia Urbinati, Sabine Geiger, et al.
Blood|March 4, 2015
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cellsMegan D Hoban, Gregory J Cost, Matthew C Mendel, et al.
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