Allan Jacobson, Ph.D., of the University of Massachusetts Medical School and co-founder of PTC Therapeutics, the company that developed ataluren, and David Bedwell, Ph.D., professor of the UAB Department of Biochemistry and Molecular Genetics, have sought to understand precisely how ataluren allows the
ribosome, the machinery of cellular protein synthesis, to skip over these inserted stop signs and produce proteins that have
normal or near -
normal function.
As expected, they found that the
normal and extra-long genetic instructions were both translated into huntingtin proteins when they met up with a
ribosome (the chef from our analogy above).