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, genetics and genomics have become increasingly reliant on computational tools that can sift through massive...