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Article | "The 60-year evolution of lipid nanoparticles for nucleic acid delivery" in Nature Reviews Drug Discovery

Published: 11 July 2024

Discover how lipid are transforming gene therapy in a new article co-authored by Pieter Cullis, Professor, Biochemistry and Molecular Biology at The University of British Columbia.

Article abstract: "Delivery of genetic information to the interior of target cells in vivo has been a major challenge facing gene therapies. This barrier is now being overcome, owing in part to dramatic advances made by lipid-based systems that have led to lipid nanoparticles (LNPs) that enable delivery of nucleic acid-based vaccines and therapeutics. Examples include the clinically approved COVID-19 LNP mÏ㽶ÊÓƵ vaccines and Onpattro (patisiran), an LNP small interfering Ï㽶ÊÓƵ therapeutic to treat transthyretin-induced amyloidosis (hATTR). In addition, a host of promising LNP-enabled vaccines and gene therapies are in clinical development. Here, we trace this success to two streams of research conducted over the past 60 years: the discovery of the transfection properties of lipoplexes composed of positively charged cationic lipids complexed with nucleic acid cargos and the development of lipid nanoparticles using ionizable cationic lipids. The fundamental insights gained from these two streams of research offer potential delivery solutions for most forms of gene therapies."

Read the full article, "The 60-year evolution of lipid nanoparticles for nucleic acid delivery", published in Nature Reviews Drug Discovery:

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