Article
All-In-One Dendrimer-Based Lipid Nanoparticles Enable Precise HDR-Mediated Gene Editing In Vivo.
Advanced materials (Deerfield Beach, Fla.) - 1 Jul 2021
Farbiak Lukas, Cheng Qiang, Wei Tuo, Álvarez-Benedicto Ester, Johnson Lindsay T, Lee Sang, Siegwart Daniel J
Abstract excerpt
Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated (Cas) protein gene editing is poised to transform the treatment of genetic diseases. However, limited progress has been made toward precise editing of DNA via homology-directed repair (HDR) that requires careful orchestration of complex steps. Herein, dendrimer-based lipid nanoparticles (dLNPs) are engineered to co-encapsulate and...
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