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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Detalles Bibliográficos
Publicado en:Vision Research Vol. 48; no. 3; pp. 453 - 470
Autores principales: 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
Formato: review Journal Article
Publicado: Pergamon Press - An Imprint of Elsevier Science Feb2008
Acceso en línea:Ver este registro en EBSCOhost
Descripción
Sumario: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.