Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo

In vivodelivery is a major barrier to the use of molecular tools for gene modification. Here we demonstrate site-specific gene editing of human cells in vivo in hematopoietic stem cell-engrafted NOD-scid IL2rγ(null) mice, using biodegradable nanoparticles loaded with triplex-forming peptide nucleic...

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Detalhes bibliográficos
Main Authors: McNeer, Nicole A., Schleifman, Erica B., Cuthbert, Amy, Brehm, Michael, Jackson, Andrew, Cheng, Christopher, Anandalingam, Kavitha, Kumar, Priti, Shultz, Leonard D., Greiner, Dale L., Saltzman, W. Mark, Glazer, Peter M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2012
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3713483/
https://ncbi.nlm.nih.gov/pubmed/23076379
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2012.82
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