Crispr’s ability to cut genetic code like scissors has just started to turn into medicines. Now, gene editing pioneer Jennifer Doudna wants to build an entire ecosystem to bring these treatments mains...

Wang, Li, et al. apply a multi-omic framework to human retinal pigment epithelium and choroid, the earliest tissues affected in age-related macular degeneration. Using a combination of single-cell transcriptomics, single-cell epigenomics, enhancer mapping, and functional testing, they evaluate nearly 2,000 variants linked to this disease and nominate dozens as pathogenic.

Careful selection of Cas13 variants and delivery methods minimizes collateral RNA degradation.

Non-protein-coding genes have been linked to a hereditary condition, retinitis pigmentosa, that causes progressive blindness.

A new in vivo genome-editing system.

TnpB is a compact RNA-guided endonuclease and evolutionary ancestor of CRISPR-Cas12 that offers a promising platform for genome engineering. However, the genome-editing activity of TnpBs remains limit...

Integrated single-cell and spatial data analysis, combined with bidirectional CRISPR screens, identify the transcription factor GLIS3 as a key driver of chronic inflammation and fibrosis and a potential marker of disease severity in patients with ulcerative colitis.

Susan Monarez is part of Nature’s 10, a list of people who shaped science in 2025.