Gene therapy interventions can cure congenital deafness

The British Journal of Natural Medicine released Feb. 4, scientists at Rosalind Franklin University of Medicine and Science in the United States have recovered some of their hearing and balance in deaf mice. heavy.

By applying gene therapy, scientists have opened a new direction for treating Usher syndrome, congenital deafness that is always accompanied by blindness.

The study published in this journal focuses on the USH1C gene related to type 1 Usher syndrome. This gene controls the "harmonin" protein that plays an important role in cochlear hair cells, specializing in the response to sound waves. and transmit electronic signals to the brain.

The researchers implanted a small strand of genetic material called " antisense oligonucleotide " into the genetically modified newborn cochlea to "turn off" the defective version of the gene that produced the "harmonin" protein form .

Only a single implant has recovered part of the hearing at very low frequencies, and also reduced symptoms of head swings due to unbalance. Some mice grew cochlear hair. This result lasts up to several months, proving that it can cure congenital deafness if early intervention with gene therapy.

Last month, doctors at Massachusetts Eye and Ear Hospital and at Harvard Medical School of Medicine reported success in using gene therapy to turn cochlear cells into hair cells.

In 2012, US University of California investigators identified the fault version of the gene VGUT3, which controls a protein that plays a crucial role in the function of receiving hair cells. cochlear.