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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Bibliografske podrobnosti
izdano v:Mol Ther Methods Clin Dev
Main Authors: 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
Format: Artigo
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2018
Teme:
Article
Online dostop: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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