Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

Adenosine deaminase (ADA)-deficient mice and healthy rhesus monkeys were studied to determine the impact of age at treatment, vector dosage, dosing schedule, repeat administration, biodistribution, and immunogenicity after systemic delivery of lentiviral vectors (LVs). In Ada−/− mice, neonatal treat...

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Bibliographic Details
Main Authors: Denise A. Carbonaro-Sarracino, Alice F. Tarantal, C. Chang I. Lee, Michael L. Kaufman, Stephen Wandro, Xiangyang Jin, Michele Martinez, Danielle N. Clark, Krista Chun, Colin Koziol, Cinnamon L. Hardee, Xiaoyan Wang, Donald B. Kohn
Format: Article
Language:English
Published: Elsevier 2020-03-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050119301299

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