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Gene therapies for inherited retinal disorders

Published online by Cambridge University Press:  20 June 2014

G. JANE FARRAR ,
SOPHIA MILLINGTON-WARD ,
NAOMI CHADDERTON ,
FIONA C. MANSERGH  and
ARPAD PALFI
G. JANE FARRAR*
Affiliation:
Smurfit Institute of Genetics, School of Genetics and Microbiology, Trinity College Dublin, Dublin 2, Ireland
SOPHIA MILLINGTON-WARD
Affiliation:
Smurfit Institute of Genetics, School of Genetics and Microbiology, Trinity College Dublin, Dublin 2, Ireland
NAOMI CHADDERTON
Affiliation:
Smurfit Institute of Genetics, School of Genetics and Microbiology, Trinity College Dublin, Dublin 2, Ireland
FIONA C. MANSERGH
Affiliation:
Smurfit Institute of Genetics, School of Genetics and Microbiology, Trinity College Dublin, Dublin 2, Ireland
ARPAD PALFI
Affiliation:
Smurfit Institute of Genetics, School of Genetics and Microbiology, Trinity College Dublin, Dublin 2, Ireland
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Abstract

Significant advances have been made over the last decade or two in the elucidation of the molecular pathogenesis of inherited ocular disorders. In particular, remarkable successes have been achieved in exploration of gene-based medicines for these conditions, both in preclinical and in clinical studies. Progress in the development of gene therapies targeted toward correcting the primary genetic defect or focused on modulating secondary effects associated with retinal pathologies are discussed in the review. Likewise, the recent utilization of genes encoding light-sensing molecules to provide new functions to residual retinal cells in the degenerating retina is discussed. While a great deal has been learned over the last two decades, the next decade should result in an increasing number of preclinical studies progressing to human clinical trial, an exciting prospect for patients, those active in research and development and bystanders alike.

Keywords

Gene therapyRetinal degenerationsAdeno-associated virusRNA interferenceOptogenetics
Type
Review Articles
Information
Visual Neuroscience , Volume 31 , Issue 4-5: Strategies for Restoring Sight in Retinal Dystrophies , September 2014 , pp. 289 - 307
Copyright
Copyright © Cambridge University Press 2014 

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