Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment
By Jocelyn Kaiser,
Science
| 12. 22. 2020
It’s troubling news that gene therapy researchers have long anticipated: A hemophilia patient injected with a virus carrying a therapeutic gene in a clinical trial has developed a liver tumor. The U.S. Food and Drug Administration (FDA) has halted the associated clinical trials, and uniQure, the Dutch firm behind the studies, is now investigating whether the virus itself caused the cancer.
Gene therapy experts say that’s unlikely. The patient had underlying conditions that predisposed him to liver cancer. Still, scientists say it’s crucial to rule out any role for adeno-associated virus (AAV), the viral delivery system, or vector, that is used in hundreds of other gene therapy trials. “Everyone will want to know what happened,” says physician-scientist David Lillicrap of Queen’s University, a hemophilia researcher who was not involved with the uniQure study.
Gene therapy for various forms of the blood-clotting disorder hemophilia has been one of the field’s latest success stories. UniQure’s hemophilia B treatment appears to be among the treatments working, with 52 of 54 patients no longer needing injections of factor IX after 6 months in its...
Related Articles
GeneWatch UK has prepared a briefing on the genetic modification of nature for the International Union for Conservation of Nature (IUCN) Congress in October 2025
The upcoming Congress claims to be “where the world comes together to set priorities and drive conservation and sustainable development action.” A major concern for those on the outside is that the Congress may advance plans to develop and encourage the use of synthetic biology in nature conservation. This could at first glance sound like...
By Aaron Ginn, The Washington Post | 09.12.2025
Earlier this year, I had dinner in D.C. with Jensen Huang, the president and chief executive of Nvidia. At one point, he said something that struck me: “Why is everyone here so negative?”
He wasn’t referring to the economy...
By Roni Caryn Rabin, The New York Times | 08.25.2025
Scientists have dreamed for centuries about using animal organs to treat ailing humans. In recent years, those efforts have begun to bear fruit: Researchers have begun transplanting the hearts and kidneys of genetically modified pigs into patients, with varying degrees...
The Center for Genetics and Society is delighted to recommend the current edition of GMWatch Review – Number 589. UK-based GMWatch, a long-standing ally, was founded in 1998 by Jonathan Matthews as an independent organization seeking to counter the enormous corporate political power and propaganda of the GMO industry and its supporters. Matthews and Claire Robinson are its directors and managing editors.
CGS works to ensure that social justice, equity, human rights, and democratic governance are front...
