Recipient organisationCentro de Investigación Médica Aplicada
Funding£10K
PeriodMar 2025 — Mar 2026
In plain English
AI plain-English summary
Around 430 million people worldwide have hearing loss, and for half of the 1-2 babies born deaf per thousand, the cause is genetic—now researchers are testing whether a gene-editing technique called CRISPR-Cas can safely correct the most common form of this inherited condition. Current gene therapies for hearing loss, such as the one for otoferlin-linked deafness now in clinical trials, deliver a working copy of a gene into inner-ear cells using a modified virus. But these effects may fade as cells divide. Gene editing, by contrast, makes permanent changes to the cell’s own DNA, potentially offering a one-time treatment that lasts a lifetime. This project addresses a critical gap: no one has yet shown that CRISPR-based editing can be delivered safely and precisely to the specific inner-ear cells responsible for hearing, nor whether it works as effectively as hoped. If successful, this research could transform the outlook for children born with genetic deafness, turning a lifelong sensory impairment into a treatable condition. Beyond the direct benefit to patients, it would establish a platform for delivering gene-editing tools to other hard-to-reach tissues in the body, with potential applications across a range of inherited disorders.
View original technical description
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. Aims 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.
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