A research team led by Juan Carlos Izpisua Belmonte, and including co–first authors Hsin-Kai (Ken) Liao and Fumiyuki Hatanaka, developed a new version of the CRISPR/Cas9 gene-editing tool, allowing them to activate genes without creating DNA breaks. This breakthrough could circumvent a major hurdle to using gene editing in the treatment of human diseases. Most CRISPR/Cas9 systems create double-stranded DNA breaks, but many researchers are opposed to creating these breaks in humans because they can cause additional health problems. However, the system developed in the Belmonte lab alters gene expression without actually breaking DNA. The team used this new approach to treat several diseases in mouse models, including diabetes, acute kidney disease and muscular dystrophy. They are now working to improve the precision of their system and apply it to more cell types in the hopes of treating a wider range of human diseases, rejuvenating specific organs and reversing age-related conditions, such as hearing loss and macular degeneration.
- Taking on the Big FiveCancer is not like other diseases. Most conditions have external causes—bacteria, viruses, injury—but cancer comes from inside us. Cells go rogue, divide recklessly, invade other tissues and spread throughout the body. They do things normal cells cannot do.
- Dan Lewis – Intense ConnectionFew trustees have had a connection as intensely personal as new Board Chair Dan Lewis, who knows firsthand that cures, indeed, begin with Salk. Thanks to the research of Salk Professor Tony Hunter, the drug Gleevec was born. And thanks to Gleevec, Lewis survived leukemia.
- Jared SmithNeuroscientist and self-described history geek Jared Smith wants to boldly go where no one has gone before. “If this were the 1400s,” asks Smith, “and we were Europeans exploring the world, where is the new world?” For Smith and colleagues in Xin Jin’s lab, the answer is simple: the brain.