Four children who were born deaf have regained almost normal hearing. Researchers at Fudan University in Shanghai announced this in a press conference… Already a subscriber? Login here!
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Four children who were born deaf have regained almost normal hearing. Researchers from Fudan University in Shanghai announced this at a press conference in Brussels at the end of October. For the first time, sensory perception was given back to a human being through genetic modification. “This is a huge achievement and a world first.” It gives us hope that one day we will be able to treat other genetic causes of deafness,” commented Natalie Loundon, head of the pediatric audiophonology department at Necker-Enfants Malades Hospital in Paris. As Le Figaro reports, the children had a very rare genetic mutation that resulted in a deficiency of otoferlin, a protein essential for communication between sensory hair cells and the auditory nerve in the inner ear. Adding a rescue gene to these cells via a harmless viral vector restored the children’s hearing after just a few weeks.
THE VIRUS
Injecting a viral vector into the inner ear, a delicate procedure, has the advantage of confining the product to the small enclosed space of the cochlea and concentrating its action. An important result that comes 19 years after the mutation and the otoferlin gene were identified by Christine Petit and her team at the Institut Pasteur. The procedure was tested on mice in 2018 and has now been successful in four children born deaf. Chinese researchers at Refreshgene Therapeutics in Shanghai drew on the experience of hearing specialist Zheng-Yi Chen, a researcher at Harvard’s Mass Eye and Ear Hospital in the US.
OTHER TESTS
A result that gives hope for the successful outcome of three further clinical studies using the same genetic engineering. The first study, conducted by US biotechnology company Regeneron Pharmaceuticals, has just returned a first positive result in a newborn and will include 22 children in the US, UK and Spain. The second event, supported by the American pharmaceutical company Eli Lilly, will involve 14 children in the USA and Taiwan. The third study will be launched in a few weeks by the French biotechnology company Sensorion in collaboration with medical teams from the Necker-Enfants malades hospital and researchers from the Institut Pasteur at the Institut de l’audition in Paris. Funded by the Anr Audinnove program of the French Ministry of Higher Education, Research and Innovation, it is now managed by Sensorion, which will also produce the viral vector carrying the repair gene. “Now we are just waiting for approval from the medical and ethical committees to start clinical trials in Europe. In particular, we had to carry out safety tests on the effect and the vector in mice and monkeys,” explained Natalie Loundon, who has coordinated the project since 2020, to Le Figaro.
Congenital deafness, the gene therapy revolution
Currently, severe deafness of genetic origin can be treated with a cochlear implant: about twenty electrodes are inserted into the cochlea (part of the inner ear) in contact with the auditory nerve and stimulated via an external receiver. This hearing aid was a revolution, but it is expensive and the results vary from person to person. However, there is actually a cure for deafness. “Instead, by injecting a gene that corrects thousands of auditory cells at the same time, we can restore natural and probably permanent hearing, since these cells are not renewed throughout life,” notes Natalie Loundon to Le Figaro. However, we also need to work at the screening level: “It is now important to spread the measurement of auditory evoked potentials in maternity wards (the headphones send sounds at a precise frequency and intensity and the electrodes placed on the scalp record the electrical responses) . of the Brain, ed.). “With this technique we can detect all types of deafness at birth,” concludes Loundon.
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