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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5008245/
https://www.ncbi.nlm.nih.gov/pubmed/27626041
http://dx.doi.org/10.1038/mtm.2016.11

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5008245/
https://www.ncbi.nlm.nih.gov/pubmed/27626041
http://dx.doi.org/10.1038/mtm.2016.11

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