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 tre...

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Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Carbonaro-Sarracino, Denise A., Tarantal, Alice F., Lee, C. Chang I., Kaufman, Michael L., Wandro, Stephen, Jin, Xiangyang, Martinez, Michele, Clark, Danielle N., Chun, Krista, Koziol, Colin, Hardee, Cinnamon L., Wang, Xiaoyan, Kohn, Donald B.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6909201/
https://ncbi.nlm.nih.gov/pubmed/31871959
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.11.004
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