Volume 44 Issue 1, January 2026

Developers have married gene-modulating oligonucleotides with the targeted precision of antibodies, and the first filings using such conjugates in Duchenne muscular dystrophy are imminent.

First-in-human studies provide hope that islet replacement therapies derived from stem cells will prove safe and effective in people with type 1 diabetes, but hurdles remain.

Our understanding of the genetic mechanisms underlying rare diseases has rapidly advanced over the past decade, largely because of technological innovations. Yet clinical practice still has a strong monogenic focus, leaving many individuals undiagnosed. This Comment outlines how technological advances such as long-read sequencing should be adopted to increase multivariant testing in the clinic.

A clinical trial attempts to standardize fetal cell transplantation and highlights challenges to be addressed in upcoming studies that use stem cell-derived neuron progenitors.

DNA delivery using lipid nanoparticles results in severe toxicity in mice. However, we find that the incorporation of endogenous anti-inflammatory lipids into the lipid nanoparticles mitigates this toxicity and enables prolonged gene expression.

We introduce a method that detects viral sequences in RNA sequencing data on the basis of highly conserved proteins, enabling the detection of more than 100,000 RNA virus species. We analyzed the presence of novel viruses and host gene expression in parallel to characterize viral tropism and host immune responses in individual cells.

The mechanism of translation initiation in linear and circular mRNAs influences translation efficiency. Covalent attachment of an N⁷-methylguanosine (m⁷G) cap increases protein production from circular mRNAs in mice. Hybridization with capped endogenous RNAs also promotes protein production in cells, suggesting that this interaction might also occur between endogenous RNAs.