New drug molecules hold promise for treating rare inherited terminal childhood disease

Scientists at the University of Exeter have identified a way to “rescue” cells that have genetically mutated, paving the way to a possible new treatment for rare terminal childhood illness such as mitochondrial disease. The research, funded by the United Mitochondrial Disease Foundation in the USA, was led by Professors Matt Whiteman and Tim Etheridge. […]

New generation CRISPR technology lays foundation for therapeutics to treat a wide range of inherited ocular diseases — ScienceDaily

A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease. Published today in Nature Biomedical Engineering, the paper, titled, “Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing,” illustrates […]