Toronto, Aug. 28 -- Researchers at the University of Toronto have developed a next-generation RNA therapeutic approach that could potentially treat a wide range of genetic diseases caused by the same type of mutation.
The study, published in Science on August 27, focuses on transfer RNA, or tRNA, engineered to help cells read through premature stop signals and produce full-length proteins.
The team, led by Bowen Li, associate professor at U of T's Leslie Dan Faculty of Pharmacy, targeted "nonsense mutations", which introduce premature stop signals into genetic instructions, resulting in little or no functional protein. Such mutations account for about 11 per cent of inherited genetic disorders and affect thousands of conditions, including...