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

• Hlavní autoři: Johanna Walther (Autor), Danny Wilbie (Autor), Vincent S. J. Tissingh (Autor), Mert Öktem (Autor), Heleen van der Veen (Autor), Bo Lou (Autor), Enrico Mastrobattista (Autor)
• Médium: Kniha
• Vydáno: MDPI AG, 2022-01-01T00:00:00Z.
• Témata: article
• On-line přístup: Connect to this object online.

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....