Gene therapy and stem cells unite
By James Gallagher,
BBC News
| 10. 13. 2011
Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists.
It means patients with a genetic disease could, one day, be treated with their own cells.
A study in Nature corrected a mutation in stem cells made from a patient with a liver disease.
Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed.
At the moment, stem cells created from a patient with a genetic illness cannot be used to cure the disease as those cells would also contain the corrupted genetic code.
Scientists, at the Wellcome Trust Sanger Institute and the University of Cambridge, were working on cirrhotic liver disease.
It is caused by a change to a single pair of letters, out of the six billion which make up the genetic code.
As a result, a protein which protects the body from damage, antitrypsin, cannot escape from the liver where it is made.
The illness is one of the most common genetic diseases, affecting...
Related Articles
By Grace Won, KQED [with CGS' Katie Hasson] | 12.02.2025
In the U.S., it’s illegal to edit genes in human embryos with the intention of creating a genetically engineered baby. But according to the Wall Street Journal, Bay Area startups are focused on just that. It wouldn’t be the first...
Several recent Biopolitical Times posts (1, 2, 3, 4) have called attention to the alarmingly rapid commercialization of “designer baby” technologies: polygenic embryo screening (especially its use to purportedly screen for traits like intelligence), in vitro gametogenesis (lab-made eggs and sperm), and heritable genome editing (also termed embryo editing or reproductive gene editing). Those three, together with artificial wombs, have been dubbed the “Gattaca stack” by Brian Armstrong, CEO of the cryptocurrency company...
Alice Wong, founder of the Disability Visibility Project, MacArthur Genius, liberationist, storyteller, writer, and friend of CGS, died on November 14. Alice shone a bright light on pervasive ableism in our society. She articulated how people with disabilities are limited not by an inability to do things but by systemic segregation and discrimination, the de-prioritization of accessibility, and the devaluation of their lives.
We at CGS learned so much from Alice about disability justice, which goes beyond rights...
By Adam Feuerstein, Stat | 11.20.2025
The Food and Drug Administration was more than likely correct to reject Biohaven Pharmaceuticals’ treatment for spinocerebellar ataxia, a rare and debilitating neurodegenerative disease. At the very least, the decision announced Tuesday night was not a surprise to anyone paying attention. Approval...
