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An ex vivo gene therapy approach in X-linked retinoschisis

PURPOSE: X-linked retinoschisis (XLRS) is juvenile-onset macular degeneration caused by haploinsufficiency of the extracellular cell adhesion protein retinoschisin (RS1). RS1 mutations can lead to either a non-functional protein or the absence of protein secretion, and it has been established that e...

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Detalles Bibliográficos
Autores principales: Bashar, Abu E., Metcalfe, Andrew L., Viringipurampeer, Ishaq A., Yanai, Anat, Gregory-Evans, Cheryl Y., Gregory-Evans, Kevin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Molecular Vision 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4919093/
https://www.ncbi.nlm.nih.gov/pubmed/27390514

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