Researchers injected a gene into the inner ear of mice with Usher syndrome and made them regain hearing and balance.
Treat Usher Syndrome With Gene Therapy? French researchers have tried the experiment with success. Their work, published in the PNAS, were carried out on rodents and pave the way for the development of treatments, by gene therapy, for certain genetic forms of deafness in humans.
Usher syndrome is a genetic disease that affects newborns or children. People who have it are born or grow up deaf and subsequently suffer from blindness due to retinitis pigmentosa. This disease affects between 3 to 6% of the deaf population from birth or with hearing impairments.
Inner ear
For the first time, researchers from the Institut Pasteur, Inserm and CNRS have been able to restore hearing and balance in a mouse model with Usher syndrome. To do this, a gene was injected into the inner ear of rodents: the USH1G gene, involved in the formation and maintenance of sensory cells in the inner ear.
“By a single local injection, after birth, of the USH1G gene, the researchers succeeded in restoring the structure, badly damaged from birth, of the mechanical-electrical transduction apparatus of the hair cells, and thus allowed the young mice to recover, and this in a sustainable way, partially hearing and completely balance, ”commented the researchers in a CNRS press release.
48 hours
Expression of the gene was detected as early as 48 hours after injection. This means that after this time, the production and localization of the relevant protein in the hair cells was found to be sufficient to improve hearing and balance in affected mice.
“This study constitutes an important step towards the design of clinical trials of gene therapy for a curative treatment of certain genetic forms of deafness in humans”, conclude the authors.
.
