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Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector

Ideally, somatic gene therapy should result in lifetime reversal of genetic deficiencies. However, to date, phenotypic correction of monogenic hyperlipidemia in mouse models by in vivo gene therapy has been short-lived and associated with substantial toxicity. We have developed a helper-dependent ad...

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Detalhes bibliográficos
Main Authors: Kim, In-Hoo, Józkowicz, Alicja, Piedra, Pedro A., Oka, Kazuhiro, Chan, Lawrence
Formato: Artigo
Idioma:Inglês
Publicado em: The National Academy of Sciences 2001
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC60862/
https://ncbi.nlm.nih.gov/pubmed/11687662
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.241506298
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