AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

Hepatocyte-restricted, AAV-mediated gene transfer is being used to provide sustained, tolerogenic transgene expression in gene therapy. However, given the episomal status of the AAV genome, this approach cannot be applied to pediatric disorders when hepatocyte proliferation may result in significant...

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Publicado en:Mol Ther Methods Clin Dev
Autores principales: Colella, Pasqualina, Sellier, Pauline, Costa Verdera, Helena, Puzzo, Francesco, van Wittenberghe, Laetitia, Guerchet, Nicolas, Daniele, Nathalie, Gjata, Bernard, Marmier, Solenne, Charles, Severine, Simon Sola, Marcelo, Ragone, Isabella, Leborgne, Christian, Collaud, Fanny, Mingozzi, Federico
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2018
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6299151/
https://ncbi.nlm.nih.gov/pubmed/30581888
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.11.002
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