Ontology highlight
ABSTRACT:
SUBMITTER: Marangoni D
PROVIDER: S-EPMC5008245 | biostudies-literature | 2016
REPOSITORIES: biostudies-literature
Marangoni Dario D Bush Ronald A RA Zeng Yong Y Wei Lisa L LL Ziccardi Lucia L Vijayasarathy Camasamudram C Bartoe Joshua T JT Palyada Kiran K Santos Maria M Hiriyanna Suja S Wu Zhijian Z Colosi Peter P Sieving Paul A PA
Molecular therapy. Methods & clinical development 20160316
X-linked retinoschisis (XLRS) is a retinal disease caused by mutations in the gene encoding the protein retinoschisin (RS1) and is one of the most common causes of macular degeneration in young men. Our therapeutic approach for XLRS is based on the administration of AAV8-scRS/IRBPhRS, an adeno-associated viral vector coding the human RS1 protein, via the intravitreal (IVT) route. Two Good Laboratory Practice studies, a 9-month study in New Zealand White rabbits (n = 124) injected with AAV8-scRS/ ...[more]