RPGR and RP2: Targets for the treatment of X-linked retinitis pigmentosa?

Stefan Veltel, Alfred Wittinghofer

Research output: Contribution to journalArticleScientificpeer-review

16 Citations (Scopus)

Abstract

Retinitis pigmentosa is the most important hereditary eye disease and there is currently no cure available. Although mutations were found in more than 40 genes in patients with retinitis pigmentosa, only two genes have thus far been found to be responsible for one of the most severe forms of the disease, X-linked retinitis pigmentosa. In this review, we highlight the current knowledge about the two gene products RPGR and RP2 and try to link genetic data from patients with functional data on the corresponding proteins. Based on the fact that recent gene therapeutic approaches for eye diseases are at a very promising stage, we discuss the potential of RPGR and RP2 as drug targets to treat retinitis pigmentosa.
Original languageEnglish
Pages (from-to)1239-1251
JournalExpert Opinion on Therapeutic Targets
Volume13
Issue number10
DOIs
Publication statusPublished - 2009
MoE publication typeA1 Journal article-refereed

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Retinitis Pigmentosa
Genes
Hereditary Eye Diseases
Eye Diseases
Therapeutics
Mutation
Pharmaceutical Preparations
Proteins

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Veltel, Stefan ; Wittinghofer, Alfred. / RPGR and RP2 : Targets for the treatment of X-linked retinitis pigmentosa?. In: Expert Opinion on Therapeutic Targets. 2009 ; Vol. 13, No. 10. pp. 1239-1251.
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RPGR and RP2 : Targets for the treatment of X-linked retinitis pigmentosa? / Veltel, Stefan; Wittinghofer, Alfred.

In: Expert Opinion on Therapeutic Targets, Vol. 13, No. 10, 2009, p. 1239-1251.

Research output: Contribution to journalArticleScientificpeer-review

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AU - Wittinghofer, Alfred

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AB - Retinitis pigmentosa is the most important hereditary eye disease and there is currently no cure available. Although mutations were found in more than 40 genes in patients with retinitis pigmentosa, only two genes have thus far been found to be responsible for one of the most severe forms of the disease, X-linked retinitis pigmentosa. In this review, we highlight the current knowledge about the two gene products RPGR and RP2 and try to link genetic data from patients with functional data on the corresponding proteins. Based on the fact that recent gene therapeutic approaches for eye diseases are at a very promising stage, we discuss the potential of RPGR and RP2 as drug targets to treat retinitis pigmentosa.

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JF - Expert Opinion on Therapeutic Targets

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