Machine learning models reveal that histone marks are predictive of gene expression across human cell types and highlight important nuances between natural control and the effects of CRISPR-Cas9-based ...

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 ...

Different variants of a gene, known as isoforms, can be transcribed and translated at varying levels within a cell or tissue. These isoforms are commonly a result of alternative splicing, which ...

Scientists identified 473 human genes that act as genetic “on/off switches,” shaping disease risk through tissue-specific or universal patterns regulated by DNA changes and hormones. Study: ...