Gene Therapy Could One Day Prevent Facial Birth Defects
Gene Therapy Could One Day Prevent Facial Birth Defects
Then they wanted to see if they could repair the genetic defect by adding back retinoic acid. All of the 403 chick embryos treated with retinoic acid overcame the effects and developed normally. The chicks also developed normally when dosed with a protein called "sonic hedgehog" (named for the video-game character), which they had previously shown to be involved in the development of this birth defect in mice.
"I think her work is right at the cutting edge," says John Sauk, DDS, PhD, professor and chair of oral and maxillofacial pathology at the University of Maryland in Baltimore. Fetal surgeons are already investigating possible ways to correct birth defects in the womb, and Sauk speculates that implants similar to the one Helms used could one day help correct human birth defects.
Gene Therapy Could One Day Prevent Facial Birth Defects
Then they wanted to see if they could repair the genetic defect by adding back retinoic acid. All of the 403 chick embryos treated with retinoic acid overcame the effects and developed normally. The chicks also developed normally when dosed with a protein called "sonic hedgehog" (named for the video-game character), which they had previously shown to be involved in the development of this birth defect in mice.
"I think her work is right at the cutting edge," says John Sauk, DDS, PhD, professor and chair of oral and maxillofacial pathology at the University of Maryland in Baltimore. Fetal surgeons are already investigating possible ways to correct birth defects in the womb, and Sauk speculates that implants similar to the one Helms used could one day help correct human birth defects.
Vital Information:
- Researchers say that doctors may someday be able to use a gene therapy to block the development of facial birth defects.
- So far, the researchers have been able to demonstrate similar birth defects developing in chickens that are deprived of a by-product of vitamin A. The facial defects could be reversed when the by-product, called retinoic acid, was restored. A protein called "sonic hedgehog" (named for the video-game character) also blocked the defect.
- Although researchers still have a lot to learn about how these defects arise in people, observers note this research is "right at the cutting edge."
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