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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主要な著者: 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.
フォーマット: Artigo
言語:Inglês
出版事項: The Association for Research in Vision and Ophthalmology 2012
主題:
オンライン・アクセス: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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