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Acid-degradable lipid nanoparticles enhance the delivery of mRNA

S. Zhao, K. Gao, H. Han, M. Stenzel, B. Yin, H. Song, A. Lawanprasert, JE. Nielsen, R. Sharma, OH. Arogundade, S. Pimcharoen, YJ. Chen, A. Paul, J. Tuma, MG. Collins, Y. Wyle, MG. Cranick, BW. Burgstone, BS. Perez, AE. Barron, AM. Smith, HY. Lee,...

. 2024 ; 19 (11) : 1702-1711. [pub] 20240823

Jazyk angličtina Země Anglie, Velká Británie

Typ dokumentu časopisecké články

Perzistentní odkaz   https://www.medvik.cz/link/bmc25003694

Grantová podpora
R33 U.S. Department of Health & Human Services | National Institutes of Health (NIH)
1DP1OD029517-01 U.S. Department of Health & Human Services | National Institutes of Health (NIH)
R01 MH125979 NIMH NIH HHS - United States
RO1EB029320-01A1 U.S. Department of Health & Human Services | National Institutes of Health (NIH)
1UM1AI164559 U.S. Department of Health & Human Services | National Institutes of Health (NIH)
R61DA048444-01 U.S. Department of Health & Human Services | National Institutes of Health (NIH)
UG3NS115599 U.S. Department of Health & Human Services | National Institutes of Health (NIH)
RO1MH125979-01 U.S. Department of Health & Human Services | National Institutes of Health (NIH)
1R21NS133881-01 U.S. Department of Health & Human Services | National Institutes of Health (NIH)
DP1 AG072438 NIA NIH HHS - United States

E-zdroje Online Plný text

NLK ProQuest Central od 2006-10-01 do Před 1 rokem
Health & Medicine (ProQuest) od 2006-10-01 do Před 1 rokem

Odkazy

PubMed 39179796
DOI 10.1038/s41565-024-01765-4
Knihovny.cz E-zdroje

Lipid nanoparticle (LNP)-mRNA complexes are transforming medicine. However, the medical applications of LNPs are limited by their low endosomal disruption rates, high toxicity and long tissue persistence times. LNPs that rapidly hydrolyse in endosomes (RD-LNPs) could solve the problems limiting LNP-based therapeutics and dramatically expand their applications but have been challenging to synthesize. Here we present an acid-degradable linker termed 'azido-acetal' that hydrolyses in endosomes within minutes and enables the production of RD-LNPs. Acid-degradable lipids composed of polyethylene glycol lipids, anionic lipids and cationic lipids were synthesized with the azido-acetal linker and used to generate RD-LNPs, which significantly improved the performance of LNP-mRNA complexes in vitro and in vivo. Collectively, RD-LNPs delivered mRNA more efficiently to the liver, lung, spleen and brains of mice and to haematopoietic stem and progenitor cells in vitro than conventional LNPs. These experiments demonstrate that engineering LNP hydrolysis rates in vivo has great potential for expanding the medical applications of LNPs.

Citace poskytuje Crossref.org

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