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.
Salk scientists modify CRISPR to epigenetically treat diabetes, kidney disease, muscular dystrophy
Featured Stories
Interaction + Infrastructure = Innovations in Healthy AgingSalk formula recalculates aging research, shifting focus to overall resilience and health span.
Professor Jan Karlseder named Salk’s Chief Science OfficerOn February 1, Jan Karlseder started as Salk’s new senior vice president and chief science officer (CSO).
Salk mourns the loss of Nobel Laureate Roger GuilleminThe Institute remembers the “father of neuroendocrinology” for his many contributions to science and friendship to all.
Daniel Hollern–Turning a cancer diagnosis into a career in basic researchAssistant Professor Daniel Hollern pivoted his career trajectory after a family member’s life-changing diagnosis pushed his already-curious mind off a cliff of questions—starting with, “What can I do to help him?”
Jerry Sheehan–Collaborating shoulder to shoulder with scientistsWhen you think about a person who heads an information technology department, you might not envision them working shoulder to shoulder with research scientists. But that’s exactly the approach Jerry Sheehan is taking as Salk’s new chief information officer (CIO).
Laura Mainz–Taking control after a cancer diagnosisLaura Mainz grew up in a tiny village in midwestern Germany, with just as many cows as people. But the sweeping landscape speckled with farms never swayed her toward plant biology—instead, her interest in the human body grew.
Black Association affinity group cultivates welcoming environmentThe Black Association at Salk (BAS) was one of the first affinity groups formed at Salk in 2020. Since that time, it has established itself as a welcoming space and go-to for resources for all Black Salk community members.
Support a legacy where cures begin.
Scientific discovery at the Salk Institute is made possible through your annual contributions. Your support will accelerate the pace of breakthroughs in understanding disease and pave the way to new drug therapies.
Get involved