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AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models.
Wang, Jiang-Hui; Lidgerwood, Grace E; Daniszewski, Maciej; Hu, Monica L; Roberts, Georgina E; Wong, Raymond C B; Hung, Sandy S C; McClements, Michelle E; Hewitt, Alex W; Pébay, Alice; Hickey, Doron G; Edwards, Thomas L.
Afiliação
  • Wang JH; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Level 7, 32 Gisborne Street, East Melbourne, VIC, 3002, Australia.
  • Lidgerwood GE; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Level 7, 32 Gisborne Street, East Melbourne, VIC, 3002, Australia.
  • Daniszewski M; Department of Anatomy and Physiology, The University of Melbourne, Parkville, Australia.
  • Hu ML; Department of Anatomy and Physiology, The University of Melbourne, Parkville, Australia.
  • Roberts GE; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Level 7, 32 Gisborne Street, East Melbourne, VIC, 3002, Australia.
  • Wong RCB; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Level 7, 32 Gisborne Street, East Melbourne, VIC, 3002, Australia.
  • Hung SSC; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Level 7, 32 Gisborne Street, East Melbourne, VIC, 3002, Australia.
  • McClements ME; Ophthalmology, Department of Surgery, The University of Melbourne, East Melbourne, VIC, Australia.
  • Hewitt AW; Shenzhen Eye Hospital, Shenzhen University School of Medicine, Shenzhen, China.
  • Pébay A; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Level 7, 32 Gisborne Street, East Melbourne, VIC, 3002, Australia.
  • Hickey DG; Ophthalmology, Department of Surgery, The University of Melbourne, East Melbourne, VIC, Australia.
  • Edwards TL; Department of Clinical Neurosciences, Nuffield Laboratory of Ophthalmology, University of Oxford, Oxford, UK.
Sci Rep ; 12(1): 9525, 2022 06 09.
Article em En | MEDLINE | ID: mdl-35680963
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doenças Retinianas / Terapia Genética / Distrofias Hereditárias da Córnea / Família 4 do Citocromo P450 Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doenças Retinianas / Terapia Genética / Distrofias Hereditárias da Córnea / Família 4 do Citocromo P450 Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article