Researchers at Rosalind Franklin University of Medicine and Science have found a way to use a single dose of a small, synthetic RNA-like molecule, called an antisense oligonucleotide (ASO), to prevent the onset of deafness in mice that have been engineered to have Usher Syndrome.
By giving the mice this one-time dose of ASO early in life, the scientists are able to “correct gene expression” and prevent the genetic mutation of Usher Syndrome to cause deafness. This does mean that the ASO must be given to the affected mice when they are young in order to start the treatment early in development, but it’s still a huge breakthrough.
Usher syndrome is the leading genetic cause of combined deafness and blindness in humans and this development of a treatment for mice could bring us one step closer to a treatment for humans. As one of the researchers said: “Successfully treating a human genetic disease in this animal model brings the possibility of treating patients much closer.”