Pasteurdon 2022: correcting certain deafness with gene therapy

I met Professor Christine Petit, director of the Joint Genetics and Physiology of Hearing research unit at the Institut Pasteur during a scientific symposium in the United States. By exchanging together, we both realized that the complementarity of our expertise could open up new avenues of research. says Professor Saaid Safieddine, a pioneer in gene therapy to treat certain types of deafness – arrived at the Institut Pasteur more than 25 years ago.

And to continue: since then, we have continued to collaborate first within her research unit at the Institut Pasteur and then within the Institut de l’Audition which she founded in 2019. “.

The Institut de l’Audition, a center of the Institut Pasteur, brings together research teams, ENT doctors and audiologists to forge ongoing collaboration around ambitious projects.

This institute brings out new areas of research to meet the needs of people who are deaf or hard of hearing. This structure provides France with a state-of-the-art institute like most European countries that already have an ear or hearing institute.

Very concretely, this mixing of proven scientific and technical knowledge in surgery, genetics or even virology allows the Hearing Institute to carry out cross-cutting research of excellence and to develop translational approaches for the benefit of hearing.

One of the objectives of its Technologies and gene therapy for deafness team within the Institut de l’Audition is to develop effective gene therapies for functional deafness (3 to 10% of deaf people worldwide) by replacing the missing functionality. .

For more than ten years, inner ear surgery and gene replacement have evolved and proven effective. Several projects are in development. The most advanced is now being developed by an industrialist, and a clinical trial should begin at the end of 2023.

Some deafness can affect the very structure of the ear, involving a lack of balance, as is the case for Usher syndrome. However, this concern for balance is major because it leads to falls and accidents. We are working on a gene therapy that would allow these patients to partially recover their hearing but also their balance.

Remember that gene therapy involves the correction or replacement of a defective gene. The precision of such a therapy is essential, because it must be administered only once to target thousands of cells in a tissue or an organ, and within these cells one gene among the 30,000 genes that make up the human genome.

In 2017, we succeeded in correcting these defects in a mouse model and this confirmed our desire to transpose this trial to humans. We are currently seeking to optimize the therapeutic vector to more effectively target the target cells concludes the researcher.

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Pasteurdon 2022: correcting certain deafness with gene therapy

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