Thanks to continued weekly medications, a 16-month-old girl shows no symptoms of a severe genetic disease that typically kills children before they turn two.
Researchers at the RIKEN Center for Developmental Biology will treat the first patient in its clinical trial testing an induced pluripotent stem cell-based treatment for age-related macular degeneration.
The Sloan Kettering researcher used mutagenic screening to probe genes and molecular pathways, including Toll and Hedgehog, essential to development in fruit flies and mice.