Scientists have created a method for editing baby genes right in the womb. In the future, work will allow the treatment and prevention of intractable genetic diseases before birth. This is stated in a study by scientists from the University of Health and Science of Oregon, published in the journal Nucleic Acids Research.
Researchers conducted all manipulations on mice and have not yet tested the new method in humans. Scientists introduced a specially designed synthetic molecule into the developing inner ear of embryonic mice 12 days after fertilization.
A molecule is a synthetic antisense oligonucleotide that, in the body, attaches to specific nucleic acid sequences. In the experiment, the goal was the so-called messenger – a class of molecules that affect where, when and how much genes are expressed in the cell.
As a result, scientists managed to correct the expression of the mutated gene in the mouse embryo that causes Asher syndrome – a disorder characterized by deafness and progressive loss of vision, affecting about 4 to 17 out of every 100 thousand people.
The authors of the work note that the new technique in the same form cannot be used to prevent similar genetic diseases in humans. However, a new study, combined with previous results, suggests that drug therapy can be administered through amniotic fluid in the uterus for the fetus. In addition, in the future this will create a similar method for the treatment of genetic diseases in humans.