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Deprecated: Implicit conversion from float 231.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Proc+Natl+Acad+Sci+U+S+A 2017 ; 114 (36): 9695-700 Nephropedia Template TP
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Local gene therapy durably restores vestibular function in a mouse model of Usher syndrome type 1G #MMPMID28835534
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 JP; Lustig L; Avan P; Petit C; Safieddine S
Proc Natl Acad Sci U S A 2017[Sep]; 114 (36): 9695-700 PMID28835534show ga
Hearing and balance impairments are major concerns and a serious burden for public health, but still lack an effective curative therapy. We assessed inner ear functions in a mouse model of Usher syndrome type 1, a developmental disorder characterized by profound congenital deafness and balance deficit, after local gene therapy. Viral transfer of the wild-type cDNA to the inner ear of the mutant mice shortly after birth resulted in a partial restoration of hearing and a long-lasting, almost complete, removal of the balance defect. The present results provide a basis for future clinical trials in humans.