Another gene therapy trial success!

Another gene therapy trial success! Chinese-US Gene therapy trial restores toddler's hearing. 

Only last month we shared the fantastic news that an Oxfordshire toddler has had her hearing restored as part of a pioneering gene therapy trial. Well, the good news keeps coming in this area as we’ve recently learnt of five children who were born deaf who have had their hearing restored in both ears.

Helping the signals get through

Doctors at Fudan University in Shanghai treated the children, aged between one and 11, as part of a Chinese-US gene therapy trial.

The children had an inherited condition where genetic mutations disrupt the body’s ability to make a protein called otoferlin which enables auditory signals to pass from the ear to the brain.

Within weeks of receiving the therapy, the children had gained hearing, could locate the sources of sounds, and recognised speech in noisy environments. Two were even recorded dancing to music.

How does the therapy work?

The therapy uses an inactive virus to deliver working copies of the affected gene into the inner ear. Cells in the ear then use the new material as a template to make working copies of the crucial protein, otoferlin.

Up to 60% of childhood deafness is due to genetic factors.

The children in this trial have a condition called DFNB9, which accounts for 2-8% of all congenital hearing loss. However, the hope is that this approach can be looked at for deafness caused by other genes or non-genetic causes.

Here to help

If you have any questions, or if you’re concerned about your or a family member’s hearing, we’re here to help. Please call our friendly team or email us to find out more.