Preclinical Safety Evaluation of a Recombinant AAV8 Vector for X-Linked Retinoschisis After Intravitreal Administration in Rabbits

X-linked retinoschisis (XLRS) is a retinal disease caused by mutations in the gene encoding the protein retinoschisin (RS1) and one of the most common causes of macular degeneration in young men. Currently, no FDA-approved treatments are available for XLRS and a replacement gene therapy could provid...

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書目詳細資料
發表在:Hum Gene Ther Clin Dev
Main Authors: Marangoni, Dario, Wu, Zhijian, Wiley, Henry E., Zeiss, Caroline J., Vijayasarathy, Camasamudram, Zeng, Yong, Hiriyanna, Suja, Bush, Ronald A., Wei, Lisa L., Colosi, Peter, Sieving, Paul A.
格式: Artigo
語言:Inglês
出版: Mary Ann Liebert, Inc. 2014
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Research Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC4275775/
https://ncbi.nlm.nih.gov/pubmed/25211193
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2014.067
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