Messenger RNA (mRNA) is an emerging class of therapeutic agent for the prevention and
treatment of a wide range of diseases. The recent success of the two highly efficacious …

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 …

In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

The expanding applications of nonviral genomic medicines in the lung remain restricted by
delivery challenges. Here, leveraging a high-throughput platform, we synthesize and screen …

Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent
and treat various diseases. To function in vivo, mRNA requires safe, effective and stable …

Gene-editing technologies, which include the CRISPR–Cas nucleases 1, 2, 3 and CRISPR
base editors 4, 5, have the potential to permanently modify disease-causing genes in …

RNA-based therapeutics have shown great promise in treating a broad spectrum of
diseases through various mechanisms including knockdown of pathological genes …

Abstract mRNA vaccines have evolved from being a mere curiosity to emerging as COVID-
19 vaccine front-runners. Recent advancements in the field of RNA technology, vaccinology …

Most known pathogenic point mutations in humans are C• G to T• A substitutions, which can
be directly repaired by adenine base editors (ABEs). In this study, we investigated the …

CRISPR–Cas gene editing and messenger RNA-based protein replacement therapy hold
tremendous potential to effectively treat disease-causing mutations with diverse cellular …