Page 12 - Biotechnology newsletter 2023-24

Gene therapy: A new hope for deaf individuals

Deafness and hearing loss are widespread and found in every age group,
region and country. According to WHO report, more than 1.5 billion people live
with earing loss, 430 million of them have disabling hearing loss and
approximately 34 million children are suffering from deafness. Many of the
impacts of hearing loss can be mitigated through early detection, sign
language programme and assistive technologies such as hearing aids, cochlear
implants, closed captioning for the people with hearing loss or deafness of any
age group. Although these methods have promising results, but due to its
repetitive training session, financial and societal burden creates an emotional
distress among this population. Due to this reason, many researchers globally
are now looking upon gene therapy method as a one-time solution against
deafness and hearing loss.

The work, conducted in Fudan, China, by a team co-led by Harvard
Medical School researches at Massachusetts Eye and Ear and by collaborators
at Fudan University’s Eye & ENT Hospital, has created a novel gene therapy
approach, giving five children who were born deaf the ability to hear. This
project done in December 2022, was the first to make use of gene therapy to
treat this condition. The researchers treated six children aged 1 to 7 who had a
mutation of the OTOF gene, which manufactures a protein important in
transmitting signals from the ear to the brain. Approximately 200,000 people
worldwide are deaf due to a mutation in the OTOF gene. The OTOF gene
encodes the otoferlin protein, produced by cells in a snail-shaped part of the
inner ear called the cochlea. In the cochlea, sound waves are translated into
electric pulses carried by nerve cells to the brain, where they are interpreted as
sound. Otoferlin plays a role in transmitting pulses from cochlear cells to the
nerves and without it, sound is translated into electric signals but never reach
the brain.

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