The efficacy of nucleic acid therapeutics (NATs) such as antisense oligonucleotides (ASOs) and small interfering RNAs relies on multiple stages of extra- and intracellular trafficking. Assessing uptak...

Antisense oligonucleotides (ASOs) can modulate gene expression at the mRNA level, providing the ability to tackle conventionally undruggable targets and usher in an era of personalized medicine. A key...

Synthetic biologists have the know-how and ambition to retool whole genomes. But the hidden complexity of biological systems continues to surprise them.

The move halts a large slice of money for most universities and research institutions virtually overnight, imperiling vital research in everything from cancer to heart disease.

Paper on hydroxychloroquine led by French researcher Didier Raoult is second-most-cited study ever to be withdrawn.

Nucleic acids have emerged as a powerful class of therapeutics. Through simple base pair complementarity, nucleic acids allow the targeting of a variety of pathologically relevant proteins and RNA molecules. However, despite the preliminary successes of nucleic acids as drugs in the clinic, limited biodistribution, inadequate delivery mechanisms, and target engagement remain key challenges in the field. A key area of research has been the chemical optimization of nucleic acid backbones to significantly enhance their “drug-like” properties. Alternatively, this review focuses on the next generation of nucleic acid chemical modifications: covalent biochemical conjugates. These conjugates are being applied to improve the delivery, functionality, and targeting. Exploiting research on heterobifunctionals, such as PROTACs, RIBOTACs, molecular glues, etc., has the potential to dramatically expand nucleic acid drug functionality and target engagement capabilities. Such next-generation chemistry-based enhancements have the potential to unlock nucleic acids as effective and versatile therapeutic agents.

Nature Medicine asks leading researchers to name their top clinical trial for 2025, from gene therapies for prion disease and sickle-cell disease to digital tools for cancer and mental health.

This 16-part series does justice to Gabriel García Márquez’s magical-realist masterwork – although the novel’s sexual politics have aged badly

Alt: a man in a blue suit and white turtleneck is standing next to a car screaming "NOOO!".

Cassava Sciences said that its drug did not significantly reduce cognitive decline in 1,900 people with mild to moderate Alzheimer’s disease.

Researchers say the social-media platform — an alternative to X — offers more control over the content they see and the people they engage with.

HOW DOES GIVINOSTAT WORK? Explained by Annemieke Aartsma-Rus.