Kathmandu. For the first time in human history, scientists have successfully restored hearing from patients born with congenital deafness using genetic therapy.
In clinical trials across the US, China and Europe, children and young people who are deaf due to a single defective gene are able to hear sounds, music and the subtle sounds of everyday life. Experts describe it as one of the most extraordinary medical breakthroughs in the last decade.
Method of treatment: How does it work?
This revolutionary treatment works not only on the symptoms of the disease, but also on the root itself.
Problem: Most hereditary hearing loss is caused by a change in the OTOF gene. This gene produces the otoferlin protein. This protein converts sound vibrations in the cells of the inner ear into electrical signals sent to the brain. In the absence of otoferlin, sound signals cannot reach the brain.
Treatment: The researchers used a safe and synthetic virus called adeno-associated virus as a carrier. The virus carries the healthy otofurlin gene and delivers it directly to the patient’s inner ear.
Results: TAG_CLOSE_span_56 Within a few weeks, cells in the inner ear begin to produce the functional otoferlin protein. This revives the process of converting sound vibrations into natural hearing.
Clinical trial results and effects{
The results of the test have been life-changing.
Resuscitation moment: Many of the patients in the trial had never heard a sound before. They began to recognize their parents’ voices, music, and simple sounds (such as footsteps or the sound of rain). Parents describe the moment their child responds to a voice for the first time as “life has been reborn”.
Success rate: TAG_CLOSE_span_53 In clinical trials involving Regeneron and other companies, 10 out of 11 children showed significant hearing improvement. Some of them regained the ability to speak in a matter of months.
{{TAG_OPEN_span_52}Importance of Early Intervention: Experts consider early intervention (early childhood treatment) to be very important in this treatment. This is because the brain’s ability to adapt to sound is strongest during the early developmental stage.
Broad meaning of gene therapy
}
The breakthrough of this treatment has the potential to not only solve deafness but also change the entire course of medical science.
{TAG_CLOSE_span_50}}
}
This technology has ushered in a new era of rooting out genetic defects, eschewing traditional methods of managing symptoms.
Possibility in other genetic diseases
}
Scientists believe that this method used to restore hearing can also be adapted to treat other hereditary diseases (such as congenital blindness, muscle weakness and some neurological problems). This simple yet powerful argument for replacing one defective gene with a healthy one can be applied to hundreds of inherited disorders.
Positive side
It restores hearing naturally without prosthetic devices (such as cochlear implants). It is a one-time treatment that relieves the hassle of managing symptoms for the rest of your life.
This surgery is less invasive than cochlear implants and has not yet seen any serious side effects.
Downside
Currently, it only treats deafness caused by a defect in a single gene. It is not useful for many hereditary causes or other types of deafness. The cost is high in the initial stages and it can be challenging to make it accessible to everyone.
It is imperative that the disease is diagnosed and treated at an early age for maximum benefit.
This breakthrough in OTOF gene therapy has opened the door to a new era for medical science. Where hereditary diseases can be treated from the root. This success has given millions of people back to the sound, connection and communication of life. The world is now moving from a cure for symptoms to a genetic purification.
