Intracellular delivery by membrane disruption: mechanisms, strategies, and concepts

MP Stewart, R Langer, KF Jensen - Chemical reviews, 2018 - ACS Publications
Intracellular delivery is a key step in biological research and has enabled decades of
biomedical discoveries. It is also becoming increasingly important in industrial and medical …

Strategies, design, and chemistry in siRNA delivery systems

Y Dong, DJ Siegwart, DG Anderson - Advanced drug delivery reviews, 2019 - Elsevier
Emerging therapeutics that utilize RNA interference (RNAi) have the potential to treat broad
classes of diseases due to their ability to reversibly silence target genes. In August 2018, the …

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 …

Optimization of lipid nanoparticle formulations for mRNA delivery in vivo with fractional factorial and definitive screening designs

KJ Kauffman, JR Dorkin, JH Yang, MW Heartlein… - Nano …, 2015 - ACS Publications
Intracellular delivery of messenger RNA (mRNA) has the potential to induce protein
production for many therapeutic applications. Although lipid nanoparticles have shown …

Current status and future perspectives on MRNA drug manufacturing

C Webb, S Ip, NV Bathula, P Popova… - Molecular …, 2022 - ACS Publications
The coronavirus disease of 2019 (COVID-19) pandemic launched an unprecedented global
effort to rapidly develop vaccines to stem the spread of the novel severe acute respiratory …

Transport by circulating myeloid cells drives liposomal accumulation in inflamed synovium

J Deprez, R Verbeke, S Meulewaeter, I Aernout… - Nature …, 2023 - nature.com
The therapeutic potential of liposomes to deliver drugs into inflamed tissue is well
documented. Liposomes are believed to largely transport drugs into inflamed joints by …

Helper lipid structure influences protein adsorption and delivery of lipid nanoparticles to spleen and liver

R Zhang, R El-Mayta, TJ Murdoch, CC Warzecha… - Biomaterials …, 2021 - pubs.rsc.org
Nucleic acids, such as messenger RNAs, antisense oligonucleotides, and short interfering
RNAs, hold great promise for treating previously 'undruggable'diseases. However, there are …

Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity

KA Whitehead, JR Dorkin, AJ Vegas, PH Chang… - Nature …, 2014 - nature.com
One of the most significant challenges in the development of clinically viable delivery
systems for RNA interference therapeutics is to understand how molecular structures …

In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

JE Dahlman, C Barnes, OF Khan, A Thiriot… - Nature …, 2014 - nature.com
Dysfunctional endothelium contributes to more diseases than any other tissue in the body.
Small interfering RNAs (siRNAs) can help in the study and treatment of endothelial cells in …

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

Y Dong, KT Love, JR Dorkin… - Proceedings of the …, 2014 - National Acad Sciences
siRNA therapeutics have promise for the treatment of a wide range of genetic disorders.
Motivated by lipoproteins, we report lipopeptide nanoparticles as potent and selective siRNA …