Topics: Genomics (Illumina)
Origin: RIIP
Project type: Service
Name of Applicant: Maxence Cornille
Date of application: 13-07-2021
Unit: Genetics and Physiology of Hearing
Location: Institut de l’audition
Phone: 0684402067
@ Mail: maxence.cornille@pasteur.fr
@ PI-Mail: saaid.safieddine@pasteur.fr
Project context and summary: Deafness and balance defects are the most prevalent inherited sensory disorder in humans and about 80% of deafness cases are attributed to a genetic cause.
In previous research project we showed AAV gene therapy partially restores hearing in several genetic deafness syndrome mouse models. We also showed that there is a linear correlation between hearing threshold rescue and the percentage of transduced sensory cells. The aim of this project is to improve this therapeutic approach by designing new AAV vectors with higher tropism for sensory cells of the inner ear.
Related team publications:Akil, O., Dyka, F., Calvet, C., Emptoz, A., Lahlou, G., Nouaille, S., Boutet de Monvel, J., Hardelin, J.-P., Hauswirth, W.W., Avan, P., Petit, C., Safieddine, S., Lustig, L.R., 2019. Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model. Proc Natl Acad Sci U S A 116, 4496–4501. https://doi.org/10.1073/pnas.1817537116
Emptoz, A., Michel, V., Lelli, A., Akil, O., Boutet de Monvel, J., Lahlou, G., Meyer, A., Dupont, T., Nouaille, S., Ey, E., Franca de Barros, F., Beraneck, M., Dulon, D., Hardelin, J.-P., Lustig, L., Avan, P., Petit, C., Safieddine, S., 2017. Local gene therapy durably restores vestibular function in a mouse model of Usher syndrome type 1G. Proc. Natl. Acad. Sci. U.S.A. 114, 9695–9700. https://doi.org/10.1073/pnas.1708894114
Tertrais, M., Bouleau, Y., Emptoz, A., Belleudy, S., Sutton, R.B., Petit, C., Safieddine, S., Dulon, D., 2019. Viral Transfer of Mini-Otoferlins Partially Restores the Fast Component of Exocytosis and Uncovers Ultrafast Endocytosis in Auditory Hair Cells of Otoferlin Knock-Out Mice. J. Neurosci. 39, 3394–3411. https://doi.org/10.1523/JNEUROSCI.1550-18.2018
Manager: marc.monot@pasteur.frStatus: Closed
