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Systematic Screening, Rational Development, and Initial Optimization of Efficacious RNA Silencing Agents for Human Rod Opsin Therapeutics

PURPOSE: To systematically evaluate human rod opsin (hRHO) mRNA for potential target sites sensitive to posttranscriptional gene silencing (PTGS) by hammerhead ribozyme (hhRz) or RNA interference (RNAi) in human cells. To develop a comprehensive strategy to identify and optimize lead candidate agent...

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Detalles Bibliográficos
Autores principales: Yau, Edwin H., Taggart, Robert T., Zuber, Mohammed, Trujillo, Alexandria J., Fayazi, Zahra S., Butler, Mark C., Sheflin, Lowell G., Breen, Jennifer B., Yu, Dian, Sullivan, Jack M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Association for Research in Vision and Ophthalmology 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6908138/
https://www.ncbi.nlm.nih.gov/pubmed/31853424
http://dx.doi.org/10.1167/tvst.8.6.28