Tyrosine-Mutant AAV8 Delivery of Human MERTK Provides Long-Term Retinal Preservation in RCS Rats

PURPOSE. The absence of Mertk in RCS rats results in defective RPE phagocytosis, accumulation of outer segment (OS) debris in the subretinal space, and subsequent death of photoreceptors. Previous research utilizing Mertk gene replacement therapy in RCS rats provided proof of concept for treatment o...

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Autors principals: Deng, Wen-Tao, Dinculescu, Astra, Li, Qiuhong, Boye, Sanford L., Li, Jie, Gorbatyuk, Marina S., Pang, Jijing, Chiodo, Vince A., Matthes, Michael T., Yasumura, Douglas, Liu, Li, Alkuraya, Fowzan S., Zhang, Kang, Vollrath, Douglas, LaVail, Matthew M., Hauswirth, William W.
Format: Artigo
Idioma:Inglês
Publicat: The Association for Research in Vision and Ophthalmology 2012
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Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3995567/
https://ncbi.nlm.nih.gov/pubmed/22408006
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1167/iovs.11-8831
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