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- W2093684383 abstract "Post-transcriptional gene silencing (PTGS) agents such as ribozymes, RNAi and antisense have substantial potential for gene therapy of human retinal degenerations. These technologies are used to knockdown a specific target RNA and its cognate protein. The disease target mRNA may be a mutant mRNA causing an autosomal dominant retinal degeneration or a normal mRNA that is overexpressed in certain diseases. All PTGS technologies depend upon the initial critical annealing event of the PTGS ligand to the target RNA. This event requires that the PTGS agent is in a conformational state able to support hybridization and that the target have a large and accessible single-stranded platform to allow rapid annealing, although such platforms are rare. We address the biocomplexity that currently limits PTGS therapeutic development with particular emphasis on biophysical variables that influence cellular performance. We address the different strategies that can be used for development of PTGS agents intended for therapeutic translation. These issues apply generally to the development of PTGS agents for retinal, ocular, or systemic diseases. This review should assist the interested reader to rapidly appreciate critical variables in PTGS development and facilitate initial design and testing of such agents against new targets of clinical interest." @default.
- W2093684383 created "2016-06-24" @default.
- W2093684383 creator A5026605437 @default.
- W2093684383 creator A5032148065 @default.
- W2093684383 creator A5040736188 @default.
- W2093684383 creator A5061168159 @default.
- W2093684383 creator A5074947833 @default.
- W2093684383 creator A5085900949 @default.
- W2093684383 date "2011-01-01" @default.
- W2093684383 modified "2023-10-16" @default.
- W2093684383 title "Variables and Strategies in Development of Therapeutic Post-Transcriptional Gene Silencing Agents" @default.
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