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 …

Two decades of research on RNA interference (RNAi) have transformed a breakthrough
discovery in biology into a robust platform for a new class of medicines that modulate mRNA …

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 …

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® …

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 …

The emergency use authorizations (EUAs) of two mRNA-based severe acute respiratory
syndrome coronavirus (SARS-CoV)-2 vaccines approximately 11 months after publication of …

Harnessing mRNA–lipid nanoparticles (LNPs) to treat patients with cancer has been an
ongoing research area that started before these versatile nanoparticles were successfully …

RNA therapeutics (RNATx) aim to treat diseases, including cancer, by targeting or
employing RNA molecules for therapeutic purposes. Amongst the most promising targets …

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 …

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 …