Lipid-based nanoparticle formulations for small molecules and RNA drugs

LM Ickenstein, P Garidel - Expert opinion on drug delivery, 2019 - Taylor & Francis
Introduction: Liposomes and lipid-based nanoparticles (LNPs) effectively deliver cargo
molecules to specific tissues, cells, and cellular compartments. Patients benefit from these …

Barcoded nanoparticles for high throughput in vivo discovery of targeted therapeutics

JE Dahlman, KJ Kauffman, Y Xing… - Proceedings of the …, 2017 - National Acad Sciences
Nucleic acid therapeutics are limited by inefficient delivery to target tissues and cells and by
an incomplete understanding of how nanoparticle structure affects biodistribution to off …

Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening

PPG Guimaraes, R Zhang, R Spektor, M Tan… - Journal of Controlled …, 2019 - Elsevier
Messenger RNA (mRNA) has recently emerged as a promising class of nucleic acid therapy,
with the potential to induce protein production to treat and prevent a range of diseases …

Augmented lipid-nanoparticle-mediated in vivo genome editing in the lungs and spleen by disrupting Cas9 activity in the liver

CD Sago, MP Lokugamage, D Loughrey… - Nature biomedical …, 2022 - nature.com
Systemically delivered lipid nanoparticles are preferentially taken up by hepatocytes. This
hinders the development of effective, non-viral means of editing genes in tissues other than …

Nanoparticle-encapsulated siRNAs for gene silencing in the haematopoietic stem-cell niche

M Krohn-Grimberghe, MJ Mitchell, MJ Schloss… - Nature biomedical …, 2020 - nature.com
Bone-marrow endothelial cells in the haematopoietic stem-cell niche form a network of
blood vessels that regulates blood-cell traffic as well as the maintenance and function of …

Optimization of a degradable polymer–lipid nanoparticle for potent systemic delivery of mRNA to the lung endothelium and immune cells

JC Kaczmarek, KJ Kauffman, OS Fenton, K Sadtler… - Nano …, 2018 - ACS Publications
mRNA therapeutics hold great potential for treating a variety of diseases through protein-
replacement, immunomodulation, and gene editing. However, much like siRNA therapy the …

Accelerating the translation of nanomaterials in biomedicine

S Mitragotri, DG Anderson, X Chen, EK Chow, D Ho… - ACS …, 2015 - ACS Publications
Due to their size and tailorable physicochemical properties, nanomaterials are an emerging
class of structures utilized in biomedical applications. There are now many prominent …

Boosting intracellular delivery of lipid nanoparticle-encapsulated mRNA

S Patel, N Ashwanikumar, E Robinson, A DuRoss… - Nano …, 2017 - ACS Publications
Intracellular delivery of mRNA holds great potential for vaccine1− 3 and therapeutic4
discovery and development. Despite increasing recognition of the utility of lipid-based …

mRNA delivery for therapeutic anti-HER2 antibody expression in vivo

Y Rybakova, PS Kowalski, Y Huang, JT Gonzalez… - Molecular Therapy, 2019 - cell.com
Antibody-based drugs are a leading class of biologics used to treat a variety of diseases,
including cancer. However, wide antibody implementation is hindered by manufacturing …

[HTML][HTML] Targeted siRNA lipid nanoparticles for the treatment of KRAS-mutant tumors

S Anthiya, SC Öztürk, H Yanik, E Tavukcuoglu… - Journal of Controlled …, 2023 - Elsevier
K-RAS is a highly relevant oncogene that is mutated in approximately 90% of pancreatic
cancers and 20–25% of lung adenocarcinomas. The aim of this work was to develop a new …