Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases

Nonintegrative AAV-mediated gene therapy in the liver is effective in adult patients but faces limitations in pediatric settings because of episomal DNA loss during hepatocyte proliferation. Gene targeting is a promising approach as it results in the permanent modification of the genome. We previous...

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Библиографические подробности
Опубликовано в: :JCI Insight
Главные авторы: De Caneva, Alessia, Porro, Fabiola, Bortolussi, Giulia, Sola, Riccardo, Lisjak, Michela, Barzel, Adi, Giacca, Mauro, Kay, Mark A., Vlahoviček, Kristian, Zentilin, Lorena, Muro, Andrés F.
Формат: Artigo
Язык:Inglês
Опубликовано: American Society for Clinical Investigation 2019
Предметы:
Research Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC6693827/
https://ncbi.nlm.nih.gov/pubmed/31211694
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.128863
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