Effects of Pathogenic Variations in the Human Rhodopsin Gene (hRHO) on the Predicted Accessibility for a Lead Candidate Ribozyme

PURPOSE: The mutation-independent strategy for hammerhead ribozyme (hhRz) or RNA interference (RNAi)-based gene therapeutics to treat autosomal dominant diseases is predicated on the hypothesis that a single therapeutic would equivalently suppress all/most of the diverse mutant mRNAs in patients wit...

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Detalles Bibliográficos
Autores principales: Froebel, Beau R., Trujillo, Alexandria J., Sullivan, Jack M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Association for Research in Vision and Ophthalmology 2017
Materias:
Genetics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5516567/
https://www.ncbi.nlm.nih.gov/pubmed/28715844
http://dx.doi.org/10.1167/iovs.16-20877

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5516567/
https://www.ncbi.nlm.nih.gov/pubmed/28715844
http://dx.doi.org/10.1167/iovs.16-20877

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