Nucleic-acid therapeutics articles from across Nature Portfolio

A circular RNA aptamer shows therapeutic efficacy in vivo in a psoriasis model.

Since the groundbreaking discovery of RNAi more than 25 years ago, several small interfering RNA (siRNA)-based therapies that target the liver have gained approval. This Review discusses principal considerations in siRNA-based drug development, focusing on the medicinal chemistry of siRNA design, the application of informatics, delivery platforms and future directions.

mRNA with engineered poly(A) tails produces prolonged higher levels of protein.

Therapeutics that target long non-coding RNAs (lncRNAs) are promising treatments for cancer. In this Review, the authors discuss how technological advances have helped improve drug discovery pipelines for lncRNAs and overview their strengths and challenges as oncological therapeutics.

The fundamental discoveries that enabled the development of effective mRNA vaccines against COVID-19 were awarded with this year’s Nobel Prize in Medicine.

The ability to create in vivo genomic medicines for tissues other than the liver has been impeded by difficulties in delivery. Using a high-throughput platform, we developed lipid nanoparticles that can effectively deliver mRNA and CRISPR–Cas9 gene editing tools to the lungs through intratracheal administration, expanding the potential clinical uses of gene editing and mRNA-based technologies.