New Gene Editing Technique Shows Promise for Treating Genetic Liver Disease
Researchers achieve a breakthrough in correcting a rare metabolic condition in mice using a safer, temporary gene-editing method.
A Baby, a Mutation, and a Medical First: How Scientists Edited Genes Inside the Body to Save a Life
In a medical milestone that sounds like science fiction, doctors have successfully edited a baby's genes inside the body to treat a rare and deadly genetic disease—marking the first time such a personalized approach has been used in a human being.
A Smart Switch for Gene Therapy: How Scientists Are Fine-Tuning Treatments to Be Just Right
The scientific paper titled "Model-guided design of microRNA-based gene circuits supports precise dosage of transgenic cargoes into diverse primary cells" introduces an innovative approach to gene therapy, aiming to ensure that therapeutic genes are expressed at optimal levels within cells.