Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

With the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approvals for Zolgensma, Luxturna, and Glybera, recombinant adeno-associated viruses (rAAVs) are considered efficient tools for gene transfer. However, studies in animals and humans demonstrate that intramuscular (IM)...

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Dettagli Bibliografici
Pubblicato in:Mol Ther
Autori principali: Gernoux, Gwladys, Gruntman, Alisha M., Blackwood, Meghan, Zieger, Marina, Flotte, Terence R., Mueller, Christian
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2020
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7054721/
https://ncbi.nlm.nih.gov/pubmed/31982038
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.01.004
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