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
Reproductive rights have been a flashpoint in national politics for decades, with the stakes surging after the Supreme Court shredded the right to an abortion. In the current presidential campaign, the battle over abortion has swelled and morphed to encompass in vitro fertilization (IVF), which has now moved rapidly from widely accepted to partisan hot button.
This dramatic shift was highlighted by the February decision of the Alabama Supreme Court that granted personhood rights to frozen IVF embryos, signaling that...
By Sara Moretto, The Varsity | 09.22.2024
It was 2020. I was wrapping up grade nine science with a solid 60 per cent, hoping that if anyone saw my failed tests in the recycling bin, it would contribute to an air of mystery about me. This reason...
By Don Sapatkin, Managed Healthcare Executive | 09.20.2024
Gene therapy comes with the expectation that it will “cure” an expanding number of genetic disorders. If you’ve never wondered – and even if you have -- what that word actually means, four Dutch researchers have a surprise in store...
By Heidi Ledford, Nature | 09.17.2024
For most of her life, Genesis Jones’s daily routine revolved around her illness, the painful blood disorder known as sickle-cell disease. Each time she left the house, she ran through a mental checklist: did she have her pain medications...