Microfluidic technologies and devices for lipid nanoparticle-based RNA delivery

M Maeki, S Uno, A Niwa, Y Okada, M Tokeshi - Journal of Controlled …, 2022 - Elsevier
In 2021, mRNA vaccines against COVID-19 were approved by the Food and Drug
Administration. mRNA vaccines are important for preventing severe COVID-19 and returning …

Cytosolic delivery of nucleic acids: The case of ionizable lipid nanoparticles

M Schlich, R Palomba, G Costabile… - Bioengineering & …, 2021 - Wiley Online Library
Ionizable lipid nanoparticles (LNPs) are the most clinically advanced nano‐delivery system
for therapeutic nucleic acids. The great effort put in the development of ionizable lipids with …

Lipid nanoparticle technology for clinical translation of siRNA therapeutics

JA Kulkarni, D Witzigmann, S Chen… - Accounts of chemical …, 2019 - ACS Publications
Conspectus Delivering nucleic acid-based therapeutics to cells is an attractive approach to
target the genetic cause of various diseases. In contrast to conventional small molecule …

[HTML][HTML] A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing

JD Finn, AR Smith, MC Patel, L Shaw, MR Youniss… - Cell reports, 2018 - cell.com
The development of clinically viable delivery methods presents one of the greatest
challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here …

Lipid nanoparticle systems for enabling gene therapies

PR Cullis, MJ Hope - Molecular Therapy, 2017 - cell.com
Genetic drugs such as small interfering RNA (siRNA), mRNA, or plasmid DNA provide
potential gene therapies to treat most diseases by silencing pathological genes, expressing …

Lipid nanoparticle technology for therapeutic gene regulation in the liver

D Witzigmann, JA Kulkarni, J Leung, S Chen… - Advanced drug delivery …, 2020 - Elsevier
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 …

On the role of helper lipids in lipid nanoparticle formulations of siRNA

JA Kulkarni, D Witzigmann, J Leung, YYC Tam… - Nanoscale, 2019 - pubs.rsc.org
Onpattro, the first RNAi-based therapeutic to receive FDA approval, is enabled by a lipid
nanoparticle (LNP) system that facilitates siRNA delivery into the cytoplasm of target cells …

On the formation and morphology of lipid nanoparticles containing ionizable cationic lipids and siRNA

JA Kulkarni, MM Darjuan, JE Mercer, S Chen… - ACS …, 2018 - ACS Publications
Lipid nanoparticles (LNPs) containing short interfering RNA (LNP-siRNA) and optimized
ionizable cationic lipids are now clinically validated systems for silencing disease-causing …

[HTML][HTML] Bone-targeted bioengineered bacterial extracellular vesicles delivering siRNA to ameliorate osteoporosis

H Liu, H Zhang, S Wang, J Cui, W Weng, X Liu… - Composites Part B …, 2023 - Elsevier
Osteoporosis (OP), characterized by decreased bone mass and destruction of bone
microarchitecture, is the most common bone degenerative disease. Conventional treatments …

Lipid nanoparticles as delivery vehicles for inhaled therapeutics

EWX Leong, R Ge - Biomedicines, 2022 - mdpi.com
Lipid nanoparticles (LNPs) have emerged as a powerful non-viral carrier for drug delivery.
With the prevalence of respiratory diseases, particularly highlighted by the current COVID-19 …