Lipid nanoparticle technology for therapeutic gene regulation in the liver
Date
2020-06-25Author
Witzigmann, Dominik
Kulkarni, Jayesh A.
Leung, Jerry
Chen, Sam
Cullis, Pieter R.
van der Meel, Roy
Metadata
Show full item record
Documentos PDF
Summary in foreign language
Hereditary genetic disorders, cancer, and infectious diseases of the liver affect millions of people around the globe and are a major public health burden. Most contemporary treatments offer limited relief as they generally aim to alleviate disease symptoms. Targeting the root cause of diseases originating in the liver by regulating malfunctioning genes with nucleic acid-based drugs holds great promise as a therapeutic approach. However, employing nucleic acid therapeutics in vivo is challenging due to their unfavorable characteristics. Lipid nanoparticle (LNP) delivery technology is a revolutionary development that has enabled clinical translation of gene therapies. LNPs can deliver siRNA, mRNA, DNA, or gene-editing complexes, providing opportunities to treat hepatic diseases by silencing pathogenic genes, expressing therapeutic proteins, or correcting genetic defects. Here we discuss the state-of-the-art LNP technology for hepatic gene therapy including formulation design parameters, production methods, preclinical development and clinical translation.
Palabras clave
Gene therapy; liver; lipid nanoparticle (LNP); lipids; hepatocyte; small interfering RNA (siRNA); messenger RNA (mRNA); DNA; guide RNA (gRNA); CRISPR/Cas9; gene silencing; gene expression; gene editingLink to resource
https://www.sciencedirect.com/science/article/pii/S0169409X20300727?via%3Dihub#ks0005Collections
Estadísticas Google Analytics
Comments
Respuesta Comentario Repositorio Expeditio
Gracias por tomarse el tiempo para darnos su opinión.