This is an Innovation Seed Fund awarded to Dr Carmen Unzu at Fundación para la Investigación Médica Aplicada, Spain. It started in April 2025.
Background
Hearing loss is the most common sensory loss, with around 430 million people affected worldwide. Around 1-2 babies out of every thousand born each year have some degree of hearing loss, and for half of them, their hearing loss is genetic. It might be possible to treat some types of genetic hearing loss using gene-based therapies.
A gene therapy for otoferlin-linked hearing loss, a genetic form of hearing loss, is currently being tested in clinical trials. It uses a small virus which is modified to deliver the gene therapy to cells in the inner ear without being able to infect any other cells. The initial results suggest that children in the trials are gaining the ability to hear after treatment, which is a big step forward for the use of gene therapy to treat hearing loss.
Another method that is showing promise in genetic therapies is gene editing. Unlike traditional gene therapies, which carry a new version of a gene into a cell to treat a condition, gene editing makes direct changes to the cell’s own DNA, meaning that the changes will likely persist for the lifetime of the cell and may therefore be more effective than traditional gene therapies
Aim
In this project, the researchers will study the safety and effectiveness of a gene editing method based on a technique called CRISPR-Cas to treat the most common form of genetic hearing loss and develop systems to deliver the gene editing technology to specific inner ear cells as effectively as possible.
Benefit
This project will advance the development of gene-based treatments for hearing loss and could ultimately benefit many people affected by genetic hearing loss.