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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...

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Detalles Bibliográficos
Autores principales: Cheng, Qiang, Wei, Tuo, Farbiak, Lukas, Johnson, Lindsay T., Dilliard, Sean A., Siegwart, Daniel J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
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