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Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice.
Marangoni, Dario; Bush, Ronald A; Zeng, Yong; Wei, Lisa L; Ziccardi, Lucia; Vijayasarathy, Camasamudram; Bartoe, Joshua T; Palyada, Kiran; Santos, Maria; Hiriyanna, Suja; Wu, Zhijian; Colosi, Peter; Sieving, Paul A.
Affiliation
  • Marangoni D; National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, Maryland, USA; Department of Biotechnology and Applied Clinical Science, University of L'Aquila, L'Aquila, Italy.
  • Bush RA; National Institute on Deafness and Other Communication Disorders, National Institutes of Health , Bethesda, Maryland, USA.
  • Zeng Y; National Institute on Deafness and Other Communication Disorders, National Institutes of Health , Bethesda, Maryland, USA.
  • Wei LL; National Eye Institute, National Institutes of Health , Bethesda, Maryland, USA.
  • Ziccardi L; National Institute on Deafness and Other Communication Disorders, National Institutes of Health , Bethesda, Maryland, USA.
  • Vijayasarathy C; National Institute on Deafness and Other Communication Disorders, National Institutes of Health , Bethesda, Maryland, USA.
  • Bartoe JT; MPI Research , Mattawan, Michigan, USA.
  • Palyada K; MPI Research , Mattawan, Michigan, USA.
  • Santos M; National Institute on Deafness and Other Communication Disorders, National Institutes of Health , Bethesda, Maryland, USA.
  • Hiriyanna S; National Eye Institute, National Institutes of Health , Bethesda, Maryland, USA.
  • Wu Z; National Eye Institute, National Institutes of Health , Bethesda, Maryland, USA.
  • Colosi P; National Eye Institute, National Institutes of Health , Bethesda, Maryland, USA.
  • Sieving PA; National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, Maryland, USA; National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.
Mol Ther Methods Clin Dev ; 5: 16011, 2016.
Article in En | MEDLINE | ID: mdl-27626041

Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Prognostic_studies Language: En Journal: Mol Ther Methods Clin Dev Year: 2016 Type: Article Affiliation country: Italy

Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Prognostic_studies Language: En Journal: Mol Ther Methods Clin Dev Year: 2016 Type: Article Affiliation country: Italy