Mutations (blue) in U2 snRNA cause a neurodevelopmental disorder about one-fifth as common as the RNU4-2/ReNU disorder, which is linked to mutations (orange) in U4 snRNA. snRNAs are shown in black, ...

LBIO-115 demonstrates robust exon 51 skipping resulting in high levels of dystrophin restoration in patient-derived muscle cells and an in vivo DMD disease model Cas13d Multi-targeting efficiently ...

Using single-nuclei RNA sequencing (snRNA-seq), scientists from Mass General Brigham, SUNY Upstate Medical University, and elsewhere have identified specific brain cell types in the hippocampus that ...

This process enables a single gene to produce multiple proteins; over evolutionary timescales, splicing can also change the size and content of genes and proteins, when different exons become included ...