The ideal targeted delivery vehicle
Lipid nanoparticles (LNPs) are well suited as delivery vehicles in gene therapy. Modulating genes has become common practice in disease research both for understanding and treating genetic ailments. In research, modulating the expression of genes can help identify the proteins involved in a disease. As a treatment, delivering a nucleic acid to suppress protein expression or replace a disease-causing protein can reverse the disorder. The low toxicity and low immunogenicity make LNPs unique alternatives to viral and cationic gene delivery systems.
The main obstacle to gene delivery is that the therapeutic genetic material becomes degraded once injected or applied systemically to an organism. Furthermore, nucleic acids are large molecular constructs that cannot be easily endocytosed or passively absorbed by cells. To overcome these challenges, LNPs have been developed to encapsulate and protect nucleic acids at high encapsulation efficiency. Also, LNPs are broadly applicable because they are taken up through a nearly-ubiquitous endogenous pathway. Their low toxicity allows them to be used with challenging cell types and to be translated from in vitro to in vivo studies.
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