Technologies and gene therapy for deafness
Institut de l'Audition
To develop effective gene therapy for hearing problems via the optimization of therapeutic vectors and their delivery systems.
Thanks to the advances in human genomics achieved over the last 20 years, important progress has been made towards understanding the pathogenesis of diverse forms of congenital and acquired deafness. The identification of deafness genes, the potential targets of treatment, and the generation and functional characterization of mouse models of human deafness have increased our knowledge of the molecular physiology of auditory sensory cells. This has paved the way for the development of new therapeutic strategies. Over the last 10 years, the efforts of our group have focused on the development of gene therapy for inner ear abnormalities. The objective is to accelerate the valorization of the many discoveries or our laboratory over the last 20 years, in the form of concrete applications of benefit to affected patients. The research performed by our team is, thus, principally translational, and largely dedicated to the development of safe and effective gene therapy for hereditary or acquired deafness, delivered by a robotic system.
We are fully aware of the challenges posed by such an objective, and have thus built a multidisciplinary team, a source of mutual enrichment, composed of students, engineers, researchers, and ENT surgeons. The two principal axes of research are:
I. Gene therapy (Group leader: S. Safieddine): The aim of this work is to generate recombinant therapeutic viral vectors to target various genetic forms of deafness, and to evaluate their efficacy, specificity and innocuity.
II RobOtol: robotic assistance for otological surgery (Group Leaders: Y. Nguyen; S. Mazalaigue): The RobOtol system has entered clinical trials, and more than 50 patients have already undergone surgery with this system since 2018. The aim of this work is to optimize the operating time for the robot-assisted surgical procedure on the middle and inner ear, making it possible to simplify surgical gestures, to increase safety and to decrease the intervention time.
63 rue de Charenton, 75012, Paris
Team leader :
Saaid Safieddine
Name of co-team leader :
Yann Nguyen
Administrative Contact Name :
Danuta Oficjalska
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