A research team supported by the National Institutes of Health (NIH) has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment. The process, from diagnosis to treatment, took only six months and marks the first time the technology has been successfully deployed to treat a human patient. The technology used in this study was developed using a platform that could be tweaked to treat a wide range of genetic disorders and opens the possibility of creating personalized treatments in other parts of the body.

Registration opens today for the 67th American Society for Radiation Oncology (ASTRO) Annual Meeting, to be held at the Moscone Center in San Francisco, September 27 to October 1, 2025. Led by ASTRO President Sameer Keole, MD, FASTRO, the conference is centered around the theme of “Rediscovering Radiation Medicine and Exploring New Indications” and is expected to attract up to 10,000 oncologists, clinicians, researchers and other health care professionals from around the globe. ASTRO news briefings will feature noteworthy and high-impact research presented at the meeting; briefing details will be announced in early September.

A team of scientists from Keck School of Medicine of USC and USC Viterbi School of Engineering has been awarded an $8 million, five-year grant from one of the National Institutes of Health to launch a Superfund Research Program Center where they will study environmental contamination from “forever chemicals,” or PFAS, which are used to make multiple household items from cookware to furniture. With the grant, the USC scientists are establishing the Southern California Superfund Research and Training Program for PFAS Assessment, Remediation and Prevention, or ShARP Center. There, they will investigate how these chemicals impact liver health, and how to detect and remove them from public water sources. Scientists at the ShARP Center will also focus on understanding how PFAS affect liver health, building on early evidence from animal models. To explore how PFAS exposure affects human liver cells, Chatzi and her team will employ advanced techniques such as 3D spheroid modeling. These lab-grown clusters of cells help scientists more accurately simulate what happens in the human body, compared to traditional 2D cell cultures. They will also conduct a population study that examines the link between PFAS exposure and liver disease in youth, a group that faces an outsized and growing risk of the condition. One major problem is PFAS contamination of public drinking water, which affects an estimated 200 million people nationwide. Researchers from the USC Viterbi School of Engineering will explore ways to remove PFAS from polluted water, including through the use of special microbes, chemicals or heating methods that can break down the chemicals.