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10.1073/pnas.1708894114

http://scihub22266oqcxt.onion/10.1073/pnas.1708894114
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C5594693!5594693!28835534
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suck abstract from ncbi


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pmid28835534      Proc+Natl+Acad+Sci+U+S+A 2017 ; 114 (36): 9695-700
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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.
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