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Bottlenecks in development of retinal therapeutic post-transcriptional gene silencing agents.

Development of post-transcriptional gene silencing (PTGS) agents for therapeutic purposes is an immense challenge in modern biology. Established technologies used to knockdown a specific target RNA and its cognate protein: antisense, ribozyme, RNAi, all conditionally depend upon an initial, critical...

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Published in:Vision Research Vol. 48; no. 3; pp. 453 - 470
Main Authors: Sullivan JM, Yau EH, Taggart RT, Butler MC, Kolniak TA, Sullivan, Jack M, Yau, Edwin H, Taggart, R Thomas, Butler, Mark C, Kolniak, Tiffany A
Format: review Journal Article
Published: Pergamon Press - An Imprint of Elsevier Science Feb2008
Online Access:View this record in EBSCOhost
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      pub: Pergamon Press - An Imprint of Elsevier Science
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        atl: Bottlenecks in development of retinal therapeutic post-transcriptional gene silencing agents.
      aug:
        au:
          Sullivan JM
          Yau EH
          Taggart RT
          Butler MC
          Kolniak TA
          Sullivan, Jack M
          Yau, Edwin H
          Taggart, R Thomas
          Butler, Mark C
          Kolniak, Tiffany A
        affil: Department of Ophthalmology, SUNY, University at Buffalo, Veterans Administration Western New York Healthcare System, Medical Research, Building 20, Room 245, 3495 Bailey Avenue, Buffalo, NY 14215, USA
      sug:
        subj:
          Gene Therapy Methods
          Genes
          Retinal Diseases Therapy
          Gene Therapy Trends
          Genetic Techniques Methods
          Funding Source
      ab: Development of post-transcriptional gene silencing (PTGS) agents for therapeutic purposes is an immense challenge in modern biology. Established technologies used to knockdown a specific target RNA and its cognate protein: antisense, ribozyme, RNAi, all conditionally depend upon an initial, critical annealing event of the PTGS ligand to a target RNA. In this review we address the nature of the bottlenecks, emphasizing the biocomplexity of target RNA structure, that currently limit PTGS therapeutic development. We briefly review existing and emerging technologies designed to release these constraints to realize the potential of PTGS agents in gene based therapies.
      pubtype: Academic Journal
      doctype:
        review
        Journal Article
      ougenre: Article
    language: English
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