March 9, 2021.
While developing a new lipid nanoparticle (LNP)-based in vivo delivery platform for CRISPR/Cas9 gene editing in the liver, Qiu et al. focused on the disruption of the Angiopoietin-like 3 (Angptl3) gene. This gene is a potential target for therapeutic interventions against human lipoprotein metabolism disorders. Using a pre-clinical mouse model, the authors were able to demonstrate specific and efficient Angptl3 disruption using their LNP delivery system, resulting in a profound reduction of low-density lipoprotein cholesterol, and triglyceride serum levels.
For more information, see: Qiu, M., et al. (2021) Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3. PNAS 118, e2020401118. DOI: 10.1073/pnas.2020401118
Keywords: CRISPR, Therapy, Cholesterol
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