Gentherapie in der Ophthalmologie: Stand der Forschung

Zusammenfassung

Der Begriff „Gentherapie“ beschreibt das Einbringen von Genen in Zellen zur Behandlung von Erkrankungen oder Gendefekten. Dazu werden Träger (Vektoren) verwendet, die diese genetischen Informationen in Zellen einschleusen. Auf diese Weise wird eine Überexpression des jeweiligen Proteins induziert oder inhibiert. Aufgrund der Möglichkeit zur visuellen Kontrolle, seines anatomischen Aufbaus und der Trennung der Blutversorgung durch die Blut-Retina-Schranke stellt das Auge ein sehr attraktives Organ für gentherapeutische Behandlungen dar. Diese Arbeit gibt einen Überblick über Fortschritte und Aussichten der Gentherapie im Fach Ophthalmologie in den letzten 10 Jahren.

Abstract

The term „gene therapy” denotes the treatment of diseases or gene deficiencies by introduction of genes into cells. To achieve this goal, vectors are used to transfer the genetic information into the cells. Thus, the protein of interest can be overexpressed or silenced. On account of its easy accessibility, the good compartmentalisation and the separation from the main bloodstream by the blood-retina barrier, the eye represents a very attractive target to treat ocular diseases by gene therapy. In this work, we provide an overview of the progress in ocular gene therapy over the last decade and give an outlook on future developments.

Schlüsselwörter

Gentherapie - Nanopartikel - Netzhaut - Hornhaut - Transduktion

Key words

gene therapy - nanoparticles - Retina - Cornea - transduction

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