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

Jack M. Sullivan; Edwin H. Yau; R. Thomas Taggart; Mark C. Butler; Tiffany A. Kolniak

doi:10.1016/j.visres.2007.09.011

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

Jack M. Sullivan
, Edwin H. Yau
, R. Thomas Taggart
, Mark C. Butler
, Tiffany A. Kolniak

Department of Ophthalmology

SUNY Buffalo

Research output: Contribution to journal › Article › peer-review

10 Scopus citations

Abstract

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.

Original language	English
Pages (from-to)	453-469
Number of pages	17
Journal	Vision Research
Volume	48
Issue number	3
DOIs	https://doi.org/10.1016/j.visres.2007.09.011
State	Published - Feb 2008

Keywords

Antisense
Gene therapy
High throughput screening
RNAi
Ribozyme
shRNA

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10.1016/j.visres.2007.09.011

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title = "Bottlenecks in development of retinal therapeutic post-transcriptional gene silencing agents",

abstract = "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.",

keywords = "Antisense, Gene therapy, High throughput screening, RNAi, Ribozyme, shRNA",

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AU - Yau, Edwin H.

AU - Taggart, R. Thomas

AU - Butler, Mark C.

AU - Kolniak, Tiffany A.

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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.

KW - Antisense

KW - Gene therapy

KW - High throughput screening

KW - RNAi

KW - Ribozyme

KW - shRNA

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M3 - Article

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AN - SCOPUS:38849116446

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VL - 48

SP - 453

EP - 469

JO - Vision Research

JF - Vision Research

IS - 3

ER -

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

Abstract

Keywords

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