In utero nanoparticle delivery for site-specific genome editing

In utero nanoparticle delivery for site-specific genome editing

The correction of genetic defects in utero could allow for improved outcomes of gene therapy. Here, the authors demonstrate safe delivery of nanoparticles to fetal mouse tissues, and show that nanoparticles containing peptide nucleic acids to edit the beta-globin gene are effective in a mouse model...

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Bibliographic Details
Main Authors: Adele S. Ricciardi, Raman Bahal, James S. Farrelly, Elias Quijano, Anthony H. Bianchi, Valerie L. Luks, Rachael Putman, Francesc López-Giráldez, Süleyman Coşkun, Eric Song, Yanfeng Liu, Wei-Che Hsieh, Danith H. Ly, David H. Stitelman, Peter M. Glazer, W. Mark Saltzman
Format: article
Language:EN
Published: Nature Portfolio 2018
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Science
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Online Access:https://doaj.org/article/adf52524b8364f4a87460fa79da2c6f9
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https://doaj.org/article/adf52524b8364f4a87460fa79da2c6f9

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