Hearing can now be restored by means of gene therapy. This has to be one of the biggest breakthroughs the 21st century has to offer. We can now cure diseases whose aetiologies originate at a genetic level.
Worldwide, gene therapy is now an option to treat genetic diseases. It has been significantly successful, giving us a newfound hope in the coming years, revolutionising the way we see healthcare treatment. A baby named Opal Sandy, was part of clinical trial to credit gene therapy as a treatment option. Her case along with patients from the UK, the US and Spain are part of this trial. Auditory neuropathy, is a disorder that disrupts hearing impulses travelling from the ear to the brain.
Gene therapy, here, is as follows: DNA is added to the modified virus, the syringe carries the modified virus, which then runs through the infusion tube to the cochlea, the gene therapy reaches the damaged hair cells, the hair cells are repaired, allowing sound to cause movement. In her case among many others, a gene called the OTOF is taken care of. The best advantage of this treatment, is that, patients no longer might need to routinely change cochlear implants to hear and this can be done at a tender age of 3.
During the period of 4 weeks, the gene therapy infusion to her right ear, started to create wonders, she can now give back a response to sound, and at 24 weeks, she was already having the ability to hear soft sounds. The type of hearing loss Opal has, is undetectable until the age of three making this procedure fruitful. Initiated in May of last year, the CHORD trial aims to answer the possibility of gene therapy as a provision of hearing for children diagnosed clinically with auditory neuropathy. The findings of her case, with others, will be exhibited at the American Society of Gene and Cell Therapy, Baltimore, USA.
The trial consists of three phases: Phase 1-low dose is received in one ear, phase 2-higher dose of gene therapy is administered in one ear after the starting dose is proved to be safe, phase 3-gene therapy is administered in both ears, with the dose being selected based on its efficacy in the previous two phases. The next five years, will consist of follow-up appointments for the patients, in order to study their speech adaptation in long term.
Nivea Vaz
Manipal College of Medical Sciences, Pokhara
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