Lipid Nanoparticle Delivery of the CRISPR/Cas9 System Directly Into the Mitochondria of Cells Carrying m.7778G>T Mutation in MtDNA (Mt-Atp8)

Lipid nanoparticle delivery of the CRISPR/Cas9 system directly into the mitochondria of cells carrying m.7778G>T mutation in MtDNA (mt-Atp8)

Mitochondrial genome mutations are associated with various diseases and gene therapy targeted to mitochondria has the potential to effectively treat such diseases. Here, we targeted a point mutation in mitochondrial DNA (mtDNA) that can cause mitochondrial diseases via delivery of the clustered, regularly interspaced, short palindromic repeats/Cas9 (CRISPR/Cas9) system to mitochondria using an innovative lipid nanoparticle (LNP) delivery system.…

The impact of lipid compositions on siRNA and mRNA lipid nanoparticle performance for pulmonary delivery - Pharma Excipients

Abstract Lipid nanoparticles (LNPs) are of significant interest as delivery systems for various RNA therapeutics, not least due to their outstanding success in applications including the COVID-19 vaccines and the siRNA therapeutic Onpattro®. As LNPs consist of different lipids, the lipid composition determines key properties of these particles. This study examines how lipid composition, especially helper and PEG-lipids, and RNA cargo (siRNA and …