Correcting Usher syndrome in utero?

May 7, 2020 Staff reporters

New research led by hearing scientists at Oregon Health & Science University suggests a way to treat and prevent intractable genetic disorders before birth.

Working with mice, researchers injected a specially designed synthetic molecule into the developing inner ear of foetal mice correcting the expression of a mutated gene that causes Usher syndrome, characterised by deafness and progressive vision loss.

The technique does not directly translate to clinical applications in people, said researchers, however the new research, combined with previous findings, suggest it may be possible to deliver the drug therapy through amniotic fluid in the womb to the foetus. The full study will be published in the journal Nucleic Acids Research in June.