gms | German Medical Science

25th Annual Meeting of the German Retina Society

German Retina Society

01.06. - 02.06.2012, Münster

Retinal gene therapy

Meeting Abstract

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  • Peter Charbel Issa - Universitäts-Augenklinik Bonn

German Retina Society. 25th Annual Conference of the German Retina Society. Münster, 01.-02.06.2012. Düsseldorf: German Medical Science GMS Publishing House; 2012. Doc12rg18

DOI: 10.3205/12rg18, URN: urn:nbn:de:0183-12rg186

This is the translated version of the article.
The original version can be found at: http://www.egms.de/de/meetings/rg2012/12rg18.shtml

Published: May 30, 2012

© 2012 Issa.
This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by-nc-nd/3.0/deed.en). You are free: to Share – to copy, distribute and transmit the work, provided the original author and source are credited.


Outline

Text

The genetic basis of inherited retinal dystrophies suggests that gene therapy is a logical therapeutic approach. It may be used to either replace or reduce the expression of defective genes. Safety and potential effectiveness have been suggested for specific disease entities (Leber’s congenital amaurosis) in first proof-of-principal clinical trials. In contrast to pharmacological treatment gene therapy has the advantage of being able to express a protein within specific cell populations and is a potentially definitive therapy. Besides replacing deficient genes in inherited diseases, additional strategies that might broaden the application of retinal gene therapy are also being developed. These include the permanent expression of neuroprotective substances or photosensitive molecules (so-called optogenetics). This overview presents an overview on current clinical trials and potential problems of retinal gene therapy.