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

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.

Afiliação

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 em En | MEDLINE | ID: mdl-27626041

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Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2016 Tipo de documento: Article País de afiliação: Itália

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