Gene therapy intervention may cure congenital deafness
Scientists at the Rosalind Franklin University of Medicine and Science in the US have partially restored hearing and balance in severely deaf mice, the British Journal of Natural Medicine reported on February 4.
By applying gene therapy, scientists have opened a new direction for treating Usher syndrome, a form of congenital deafness that is always accompanied by blindness.
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The study, published in the journal, focused on the gene USH1C, which is linked to Usher syndrome type 1. This gene controls the protein "harmonin," which plays an important role in the hair cells in the cochlea, which respond to sound waves and send electrical signals to the brain.
The researchers implanted a small strand of genetic material called an "antisense oligonucleotide" into the cochlea of newborn mice that had been genetically engineered to "turn off" the faulty version of the gene that produces forms of the "harmonin" protein.
A single implant restored some hearing at very low frequencies, and also reduced head-bobbing symptoms caused by imbalance. Some mice grew cochlear hairs. This effect lasted for several months, suggesting that congenital deafness can be cured with early intervention using gene therapy.
Last month, doctors at the Massachusetts Eye and Ear Infirmary and at Harvard Medical School in the US announced that they had also successfully used gene therapy to turn cochlear cells into hair cells.
In 2012, investigators at the University of California identified the location of a faulty version of the VGUT3 gene, which controls a protein that plays a crucial role in the signaling function of cochlear hair cells.
According to (TTXVN) - VT
