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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| Auteurs principaux: | , , , , , , , , , , , , , , , |
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| Format: | article |
| Langue: | EN |
| Publié: |
Nature Portfolio
2018
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| Sujets: | |
| Accès en ligne: | https://doaj.org/article/adf52524b8364f4a87460fa79da2c6f9 |
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