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...

Ful tanımlama

Kaydedildi:
Detaylı Bibliyografya
Yayımlandı:Mol Ther Methods Clin Dev
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group 2016
Konular:
Article
Online Erişim: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
Etiketler: Etiketle
Etiket eklenmemiş, İlk siz ekleyin!

Benzer Materyaller