Gene Therapy Restores Hearing in 90% of Patients in Landmark Trial

6 articles · Updated · Livescience.com · Apr 22

A large clinical trial in China found that gene therapy restored hearing in 90% of patients with inherited deafness caused by OTOF gene mutations.
The therapy, using an AAV vector, showed rapid and lasting improvements, with some children regaining the ability to hear whispers and develop speech.
These results, published in Nature, suggest gene therapy could become a transformative treatment for certain types of genetic hearing loss, especially in children.

With the OTOF gene conquered, how close are we to a universal therapy for all forms of inherited hearing loss?

Hearing restoration has lasted 2.5 years, but what are the unknown long-term risks of this viral gene therapy?

The therapy restored hearing in 90% of patients, but what makes the other 10% resistant to this breakthrough treatment?

How might a 'cure' for genetic deafness reshape the future of Deaf culture and sign language?

After a lifetime of silence, how does the adult brain learn to process the sudden and complex world of sound?

This 'one-and-done' cure is a medical miracle, but could its high upfront cost make it inaccessible to most?