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Impact of Formulation Conditions on Lipid Nanoparticle Characteristics and Functional Delivery of CRISPR RNP for Gene Knock-Out and Correction

The CRISPR-Cas9 system is an emerging therapeutic tool with the potential to correct diverse genetic disorders. However, for gene therapy applications, an efficient delivery vehicle is required, capable of delivering the CRISPR-Cas9 components into the cytosol of the intended target cell population....

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Detalles Bibliográficos
Autores principales: Walther, Johanna, Wilbie, Danny, Tissingh, Vincent S. J., Öktem, Mert, van der Veen, Heleen, Lou, Bo, Mastrobattista, Enrico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8778360/
https://www.ncbi.nlm.nih.gov/pubmed/35057110
http://dx.doi.org/10.3390/pharmaceutics14010213