Literature DB >> 17976683

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

Jack M Sullivan1, Edwin H Yau, R Thomas Taggart, Mark C Butler, Tiffany A Kolniak.   

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.

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Year:  2007        PMID： 17976683      PMCID： PMC3388035          DOI： 10.1016/j.visres.2007.09.011

Source DB:  PubMed          Journal:  Vision Res        ISSN： 0042-6989            Impact factor:   1.886


  52 in total

1.  Sequence elements outside the hammerhead ribozyme catalytic core enable intracellular activity.

Authors:  Anastasia Khvorova; Aurélie Lescoute; Eric Westhof; Sumedha D Jayasena
Journal:  Nat Struct Biol       Date:  2003-07-27

2.  Sfold web server for statistical folding and rational design of nucleic acids.

Authors:  Ye Ding; Chi Yu Chan; Charles E Lawrence
Journal:  Nucleic Acids Res       Date:  2004-07-01       Impact factor: 16.971

3.  Kinetic analysis of the RNAi enzyme complex.

Authors:  Benjamin Haley; Phillip D Zamore
Journal:  Nat Struct Mol Biol       Date:  2004-05-30       Impact factor: 15.369

4.  Functional heterogeneity of mutant rhodopsins responsible for autosomal dominant retinitis pigmentosa.

Authors:  C H Sung; B G Schneider; N Agarwal; D S Papermaster; J Nathans
Journal:  Proc Natl Acad Sci U S A       Date:  1991-10-01       Impact factor: 11.205

5.  Implications of ribozyme kinetics for targeting the cleavage of specific RNA molecules in vivo: more isn't always better.

Authors:  D Herschlag
Journal:  Proc Natl Acad Sci U S A       Date:  1991-08-15       Impact factor: 11.205

6.  Rapid determination and quantitation of the accessibility to native RNAs by antisense oligodeoxynucleotides in murine cell extracts.

Authors:  M Scherr; J J Rossi
Journal:  Nucleic Acids Res       Date:  1998-11-15       Impact factor: 16.971

7.  Secreted placental alkaline phosphatase: a powerful new quantitative indicator of gene expression in eukaryotic cells.

Authors:  J Berger; J Hauber; R Hauber; R Geiger; B R Cullen
Journal:  Gene       Date:  1988-06-15       Impact factor: 3.688

8.  Mutations in the human retinal degeneration slow gene in autosomal dominant retinitis pigmentosa.

Authors:  K Kajiwara; L B Hahn; S Mukai; G H Travis; E L Berson; T P Dryja
Journal:  Nature       Date:  1991-12-12       Impact factor: 49.962

Review 9.  The challenges of modeling mammalian biocomplexity.

Authors:  Jeremy K Nicholson; Elaine Holmes; John C Lindon; Ian D Wilson
Journal:  Nat Biotechnol       Date:  2004-10       Impact factor: 54.908

10.  Transgenic mice with a rhodopsin mutation (Pro23His): a mouse model of autosomal dominant retinitis pigmentosa.

Authors:  J E Olsson; J W Gordon; B S Pawlyk; D Roof; A Hayes; R S Molday; S Mukai; G S Cowley; E L Berson; T P Dryja
Journal:  Neuron       Date:  1992-11       Impact factor: 17.173
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  8 in total

1.  A cellular high-throughput screening approach for therapeutic trans-cleaving ribozymes and RNAi against arbitrary mRNA disease targets.

Authors:  Edwin H Yau; Mark C Butler; Jack M Sullivan
Journal:  Exp Eye Res       Date:  2016-05-25       Impact factor: 3.467

2.  Rapid, cell-based toxicity screen of potentially therapeutic post-transcriptional gene silencing agents.

Authors:  Tiffany A Kolniak; Jack M Sullivan
Journal:  Exp Eye Res       Date:  2011-01-21       Impact factor: 3.467

3.  Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research.

Authors:  Mark C Butler; Jack M Sullivan
Journal:  J Vis Exp       Date:  2018-11-02       Impact factor: 1.355

4.  Development of lead hammerhead ribozyme candidates against human rod opsin mRNA for retinal degeneration therapy.

Authors:  Heba E Abdelmaksoud; Edwin H Yau; Michael Zuker; Jack M Sullivan
Journal:  Exp Eye Res       Date:  2008-12-06       Impact factor: 3.467

5.  Variables and strategies in development of therapeutic post-transcriptional gene silencing agents.

Authors:  Jack M Sullivan; Edwin H Yau; Tiffany A Kolniak; Lowell G Sheflin; R Thomas Taggart; Heba E Abdelmaksoud
Journal:  J Ophthalmol       Date:  2011-06-30       Impact factor: 1.909

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

Authors:  Beau R Froebel; Alexandria J Trujillo; Jack M Sullivan
Journal:  Invest Ophthalmol Vis Sci       Date:  2017-07-01       Impact factor: 4.799

7.  Systematic Screening, Rational Development, and Initial Optimization of Efficacious RNA Silencing Agents for Human Rod Opsin Therapeutics.

Authors:  Edwin H Yau; Robert T Taggart; Mohammed Zuber; Alexandria J Trujillo; Zahra S Fayazi; Mark C Butler; Lowell G Sheflin; Jennifer B Breen; Dian Yu; Jack M Sullivan
Journal:  Transl Vis Sci Technol       Date:  2019-12-12       Impact factor: 3.283

Review 8.  Gene therapy in animal models of autosomal dominant retinitis pigmentosa.

Authors:  Brian Rossmiller; Haoyu Mao; Alfred S Lewin
Journal:  Mol Vis       Date:  2012-10-06       Impact factor: 2.367
  8 in total

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