Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

Recombinant immunotoxins represent a sophisticated class of biotherapeutics that combine the targeting specificity of antibody fragments with the potent cytotoxic effects of bacterial or plant toxins.

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...

Maoli Duan does not work for, consult, own shares in or receive funding from any company or organization that would benefit from this article, and has disclosed no relevant affiliations beyond their ...

Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Some of the year's notable breakthroughs include the first ...

Peking University, July 23, 2025: On July 14, 2025, Professor Su Xiaodong’s team from the School of Life Sciences at Peking University, in collaboration with Professor John Rasko’s team from the ...

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not without risk. When this type of “genetic surgery” is performed on DNA, for ...

Ajinomoto CELLiST Korea, a subsidiary of the Ajinomoto Group specializing in the development and manufacturing of cell culture media, has launched CELLiSTTM AAV Production Supplement 1, a newly ...