In a landmark achievement for genetic medicine, an infant named KJ Muldoon has become the first person to receive a customized CRISPR-based gene-editing therapy to treat carbamoyl phosphate synthetase 1 (CPS1) deficiency—a rare and often fatal metabolic disorder. This pioneering treatment, developed by researchers at the Children's Hospital of Philadelphia and the University of Pennsylvania, offers new hope for patients with rare genetic diseases.

Proteins are the fundamental building blocks and functional molecules of life. From catalyzing reactions to transmitting signals,...

In the era of big data biology, we are collecting enormous amounts of molecular data from human tissues — from genetic sequences to patterns of chemical modification and regulatory RNA activity. These multi-omics datasets promise unprecedented insights into disease mechanisms, especially complex diseases like cancer. But making sense of this data requires smart computational tools that can both integrate multiple biological data types and interpret what the data means . Tha