Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs

SA Dilliard, DJ Siegwart - Nature Reviews Materials, 2023 - nature.com
Genetic drugs based on nucleic acid biomolecules are a rapidly emerging class of
medicines that directly reprogramme the central dogma of biology to prevent and treat …

Unlocking the promise of mRNA therapeutics

E Rohner, R Yang, KS Foo, A Goedel… - Nature biotechnology, 2022 - nature.com
The extraordinary success of mRNA vaccines against coronavirus disease 2019 (COVID-19)
has renewed interest in mRNA as a means of delivering therapeutic proteins. Early clinical …

The role of lipid components in lipid nanoparticles for vaccines and gene therapy

CH Albertsen, JA Kulkarni, D Witzigmann… - Advanced drug delivery …, 2022 - Elsevier
Lipid nanoparticles (LNPs) play an important role in mRNA vaccines against COVID-19. In
addition, many preclinical and clinical studies, including the siRNA-LNP product, Onpattro® …

The clinical progress of mRNA vaccines and immunotherapies

AJ Barbier, AY Jiang, P Zhang, R Wooster… - Nature …, 2022 - nature.com
The emergency use authorizations (EUAs) of two mRNA-based severe acute respiratory
syndrome coronavirus (SARS-CoV)-2 vaccines approximately 11 months after publication of …

[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 …

CAR T cells produced in vivo to treat cardiac injury

JG Rurik, I Tombácz, A Yadegari… - Science, 2022 - science.org
Fibrosis affects millions of people with cardiac disease. We developed a therapeutic
approach to generate transient antifibrotic chimeric antigen receptor (CAR) T cells in vivo by …

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 …

In vivo hematopoietic stem cell modification by mRNA delivery

L Breda, TE Papp, MP Triebwasser, A Yadegari… - Science, 2023 - science.org
Hematopoietic stem cells (HSCs) are the source of all blood cells over an individual's
lifetime. Diseased HSCs can be replaced with gene-engineered or healthy HSCs through …

An ionizable lipid toolbox for RNA delivery

X Han, H Zhang, K Butowska, KL Swingle… - Nature …, 2021 - nature.com
Recent years have witnessed incredible growth in RNA therapeutics, which has benefited
significantly from decades of research on lipid nanoparticles, specifically its key component …

The current landscape of nucleic acid therapeutics

JA Kulkarni, D Witzigmann, SB Thomson… - Nature …, 2021 - nature.com
The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …