[HTML][HTML] Drug delivery systems for RNA therapeutics

K Paunovska, D Loughrey, JE Dahlman - Nature Reviews Genetics, 2022 - nature.com
RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

Chemistry of lipid nanoparticles for RNA delivery

Y Eygeris, M Gupta, J Kim, G Sahay - Accounts of chemical …, 2021 - ACS Publications
Conspectus Lipid nanoparticles (LNPs) are a type of lipid vesicles that possess a
homogeneous lipid core. These vesicles are widely used in small-molecule drug and …

Lipids and lipid derivatives for RNA delivery

Y Zhang, C Sun, C Wang, KE Jankovic… - Chemical reviews, 2021 - ACS Publications
RNA-based therapeutics have shown great promise in treating a broad spectrum of
diseases through various mechanisms including knockdown of pathological genes …

Peptide-guided lipid nanoparticles deliver mRNA to the neural retina of rodents and nonhuman primates

M Herrera-Barrera, RC Ryals, M Gautam, A Jozic… - Science …, 2023 - science.org
Lipid nanoparticle (LNP)–based mRNA delivery holds promise for the treatment of inherited
retinal degenerations. Currently, LNP-mediated mRNA delivery is restricted to the retinal …

Engineering lipid nanoparticles for enhanced intracellular delivery of mRNA through inhalation

J Kim, A Jozic, Y Lin, Y Eygeris, E Bloom, X Tan… - ACS …, 2022 - ACS Publications
Despite lipid nanoparticles'(LNPs) success in the effective and safe delivery of mRNA
vaccines, an inhalation-based mRNA therapy for lung diseases remains challenging. LNPs …

Self-assembled mRNA vaccines

J Kim, Y Eygeris, M Gupta, G Sahay - Advanced drug delivery reviews, 2021 - Elsevier
Abstract mRNA vaccines have evolved from being a mere curiosity to emerging as COVID-
19 vaccine front-runners. Recent advancements in the field of RNA technology, vaccinology …

Drug delivery systems for CRISPR-based genome editors

V Madigan, F Zhang, JE Dahlman - Nature Reviews Drug Discovery, 2023 - nature.com
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

Endosomal escape: A bottleneck for LNP-mediated therapeutics

S Chatterjee, E Kon, P Sharma… - Proceedings of the …, 2024 - National Acad Sciences
Lipid nanoparticles (LNPs) have recently emerged as a powerful and versatile clinically
approved platform for nucleic acid delivery, specifically for mRNA vaccines. A major …

Nanoparticle stereochemistry-dependent endocytic processing improves in vivo mRNA delivery

MZC Hatit, CN Dobrowolski, MP Lokugamage… - Nature Chemistry, 2023 - nature.com
Stereochemistry can alter small-molecule pharmacokinetics, safety and efficacy. However, it
is unclear whether the stereochemistry of a single compound within a multicomponent …

[HTML][HTML] Lipid nanoparticles with PEG-variant surface modifications mediate genome editing in the mouse retina

M Gautam, A Jozic, GLN Su, M Herrera-Barrera… - Nature …, 2023 - nature.com
Ocular delivery of lipid nanoparticle (LNPs) packaged mRNA can enable efficient gene
delivery and editing. We generated LNP variants through the inclusion of positively charged …