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 …

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 …

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

CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

Messenger RNA has now been used to vaccinate millions of people. However, the diversity
of pulmonary pathologies, including infections, genetic disorders, asthma and others …

In humans, lipid nanoparticles (LNPs) have safely delivered therapeutic RNA to hepatocytes
after systemic administration and to antigen-presenting cells after intramuscular injection …

Extracellular vesicles (EVs) carry diverse bioactive components including nucleic acids,
proteins, lipids and metabolites that play versatile roles in intercellular and interorgan …

Lipid nanoparticles (LNPs) are clinically proven to successfully deliver both small interfering
RNA (siRNA) therapeutics and larger mRNA payloads for prophylactic vaccine applications …

Cells that were previously described as homogeneous are composed of subsets with distinct
transcriptional states. However, it remains unclear whether this cell heterogeneity influences …

Conspectus mRNA drugs can preempt infectious disease and treat Mendelian disorders,
such as sickle cell anemia, muscular dystrophy, and cystic fibrosis, as well as autoimmunity …