CRISPR–Cas-based genome editing holds great promise for targeting genetic disorders,
including inborn errors of hepatocyte metabolism. Precise correction of disease-causing …

Programmable genome integration of large, diverse DNA cargo without DNA repair of
exposed DNA double-strand breaks remains an unsolved challenge in genome editing. We …

Base editors are RNA-programmable deaminases that enable precise single-base conversions
in genomic DNA. However, off-target activity is a concern in the potential use of base …

Prime editing is a highly versatile CRISPR-based genome editing technology that works
without DNA double-strand break formation. Despite rapid technological advances, in vivo …

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 efficacy …

Background Liraglutide 3·0 mg was shown to reduce bodyweight and improve glucose
metabolism after the 56-week period of this trial, one of four trials in the SCALE programme. In …

Forward genetic screens with genome-wide CRISPR libraries are powerful tools for resolving
cellular circuits and signaling pathways. Applying this technology to organoids, however, …

Programmable approaches to sense and respond to the presence of specific RNAs in
biological systems have broad applications in research, diagnostics, and therapeutics. Here we …

Epithelial organoids are simplified models of organs grown in vitro from embryonic and
adult stem cells. They are widely used to study organ development and disease, and enable …

Our ability to edit genomes lags behind our capacity to sequence them, but the growing
understanding of CRISPR biology and its application to genome, epigenome and transcriptome …