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Gene therapeutic approaches for dominant retinopathies.

Authors: G Jane, Farrar; Arpad, Palfi; Mary, O'Reilly;

Gene therapeutic approaches for dominant retinopathies.

Abstract

Over the past two decades, significant progress has been made in defining the molecular pathogenesis of hereditary retinal degenerations. Many of these are characterised by immense genetic heterogeneity. For example, in retinitis pigmentosa (RP), the most common form of this group of disorders, over 50 disease causing genes have been implicated, 20 of which are inherited in an autosomal dominant manner. Knowledge of the underlying genetic pathogenesis together with the availability of animal models and vectors for delivery has enabled exploration of gene-based therapies for inherited retinopathies. Notably, many studies have focused on treatment of recessive forms of these disorders and significant progress including ongoing clinical trials have been achieved. Progress in developing gene therapies for dominant retinopathies has been slower. One reason for this is that gene therapies for many dominant diseases, which are targeted to correcting the primary genetic defect, are likely to require suppression of the mutant gene. Alternative therapeutic approaches, which involve modulating secondary features associated with the disease pathology (such as ER stress or apoptosis) are also being explored. This review is focused on the development of gene-based therapies for dominantly inherited retinopathies. The main topics discussed are suppression technologies, preclinical animal models, retinal gene delivery and therapeutic strategies.

Related Organizations
Keywords

Retinal Diseases, Retinal Degeneration, Gene Transfer Techniques, Humans, Genetic Therapy, Genes, Dominant

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    14
    popularity
    This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
    Average
    influence
    This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
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    impulse
    This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
    Top 10%
Powered by OpenAIRE graph
Found an issue? Give us feedback
selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
14
Average
Average
Top 10%
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