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Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity

Immune responses to therapeutic transgenes are a potential hurdle to treat monogenic muscle disorders. These responses result from the neutralizing activity of transgene-specific B cells and cytotoxic T cells recruited upon gene transfer. We explored here how dual muscle-liver expression of a foreig...

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Pubblicato in:JCI Insight
Autori principali: Bartolo, Laurent, Li Chung Tong, Stéphanie, Chappert, Pascal, Urbain, Dominique, Collaud, Fanny, Colella, Pasqualina, Richard, Isabelle, Ronzitti, Giuseppe, Demengeot, Jocelyne, Gross, David A., Mingozzi, Federico, Davoust, Jean
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society for Clinical Investigation 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6629094/
https://ncbi.nlm.nih.gov/pubmed/31167976
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.127008
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