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Selective ORgan Targeting (SORT) nanoparticles for tissue specific mRNA delivery and CRISPR/Cas gene editing
CRISPR/Cas gene editing and messenger RNA (mRNA)-based protein replacement therapy hold tremendous potential to effectively treat disease-causing mutations with diverse cellular origin. However, it is currently impossible to rationally design nanoparticles that selectively target specific tissues. H...
Autores principales: | Cheng, Qiang, Wei, Tuo, Farbiak, Lukas, Johnson, Lindsay T., Dilliard, Sean A., Siegwart, Daniel J. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7735425/ https://www.ncbi.nlm.nih.gov/pubmed/32251383 http://dx.doi.org/10.1038/s41565-020-0669-6 |
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