Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice

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-assoc...

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Библиографические подробности
Опубликовано в: :Mol Ther Methods Clin Dev
Главные авторы: 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
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2016
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Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC5008245/
https://ncbi.nlm.nih.gov/pubmed/27626041
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.11
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