Gene editor may have cured infant of a deadly metabolic disorder
By Jocelyn Kaiser,
Science
| 01. 14. 2025
A little-known gene editor, tested with help from a disgraced gene therapist seeking redemption, may have cured a 1-year-old boy of a deadly metabolic disorder. Announced last week by a company developing the therapy, the result could be the first success at stitching a curative gene into a “safe harbor,” a specific chromosomal location where its integration is unlikely to disrupt existing DNA in a way that triggers cancer or other problems. Because the gene should now be integrated in the baby’s genome, in this case within cells of the boy’s liver, it should persist as the organ—and person—grows.
The gene editor, dubbed ARCUS, is a DNA-cutting enzyme known as a nuclease, It is in some ways simpler and potentially better than the more famous CRISPR platform and could also help treat other genetic metabolic disorders. The company, iECURE, will not present data for the treated infant until March. But the apparent success of the safe harbor approach with the editor in the very first patient who received it is especially significant for iECURE co-founder James Wilson, who helped develop...
Related Articles
By Elizabeth Dwoskin and Zoeann Murphy, The Washington Post | 10.01.2025
MEXICO CITY — When she walked into an IVF clinic in June, Alin Quintana knew it would be the last time she would try to conceive a child. She had prepared herself spiritually and mentally for the visit: She had traveled to a nearby...
By Rob Stein, NPR | 09.30.2025
Scientists have created human eggs containing genes from adult skin cells, a step that someday could help women who are infertile or gay couples have babies with their own genes but would also raise difficult ethical, social and legal issues...
By Jessica Mouzo, El País | 10.03.2025
DNA is the molecule of life: this double-helix structure, present in every cell in the body and organized into fragments called genes, stores the instructions for making organisms function. It is a highly precise biological machine, but sometimes it breaks...
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...