Gene Therapy: Editorial Control
By Katharine Gammon,
Nature News
| 11. 12. 2014
Untitled Document
Tiny changes in DNA can have huge consequences. For years, scientists have been trying to 'fix' these mutations in the hope of treating and potentially curing some of humanity's most devastating genetic diseases. After some tragic early setbacks (see Nature 420, 116–118; 2002), techniques that allow precise genetic manipulation have created a surge of research.
Although most existing treatments for genetic diseases typically only target symptoms, genetic manipulation or 'gene therapy' goes after the cause itself. The approach involves either inserting a functional gene into DNA or editing a faulty one that is already there, so the conditions most likely to prove curable are those caused by a single mutation. Sickle-cell disease is a perfect candidate: it is caused by a change in just one amino acid at a specific site in the β-globin gene. This results in the production of abnormal haemoglobin proteins that cause the red blood cells that house them to twist and become sickle shaped. The distorted cells get sticky, adhere to each other and block blood vessels, preventing oxygenated blood from...
Related Articles
By Annika Inampudi, Science | 08.01.2025
In June, Sara* received a message asking whether she wanted to continue to participate in a massive, multicenter research project led by scientists at Aarhus University in Denmark. The iPsych study, the message said, had sequenced her genetic data from...
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...
By Katherine Drabiak, Journal of Medical Ethics Forum | 08.07.2025
Adapted from Mitochondrial DNA at
National Human Genome Research Institute
Recently, media outlets around the world have been reporting on children born from pronuclear genome transfer (sometimes called “3-parent IVF,” “mitochondrial donation” or “mitochondrial replacement therapy”) at Newcastle Fertility Center...
By Annika Inampudi, Science | 07.10.2025
Before a baby in the United States reaches a few days old, doctors will run biochemical tests on a few drops of their blood to catch certain genetic diseases that need immediate care to prevent brain damage or other serious...