Zusammenfassung
In der Vergangenheit wurden euphorische Erwartungen in die Gentherapie geweckt, die
aber bisher nicht nur nicht erfüllt, sondern im Gegenteil wegen eines Ausbleibens
konkreter klinischer Erfolge sowie des Eintretens tragischer Zwischenfälle enttäuscht
wurden. Trotz des großen Zukunftspotenzials der Gentherapie werden wohl für längere
Zeit zahlreiche gentherapeutische Pilotstudien abgebrochen werden. Aber trotz aller
Misserfolge werden Krankheiten für experimentelle Protokolle, die beispielsweise nur
eine vorübergehenden Genexpression vorsehen oder solche, die sich auf einzelne Organe
beschränken und daher nur eine niedrige Dosis des Vehikels (Vektors) benötigen, weiterhin
Ziel experimenteller Gentransferversuche bleiben. Wegen seiner guten und selektiven
Erreichbarkeit, des geringen Volumens und des dadurch bedingten geringen Bedarfs an
Genfähren sowie seines besonderen immunologischen Status ist gerade das Auge als potenzielles
Zielorgan hervorragend geeignet. Die Hornhauttransplantation ist eine Indikation mit
verbesserbarer Prognose, die vom Methodenkatalog der Gentransferprotokolle profitieren
kann. Die Hornhaut steht zudem vor einer Transplantation einem Gentransfer ex vivo
zur Verfügung. Die Augenheilkunde könnte daher beim klinischen Gentransfer eine Pionierstellung
einnehmen. Eine Literatursynopse beschreibt den gegenwärtigen Stand der experimentellen
Verbesserung der Hornhauttransplantation, der Beeinflussung der Narbenbildung, der
Neovaskularisation und der Herpesinfektion der Kornea. Das wesentliche Problem der
Gentherapie ist die nicht zufrieden stellende Technik des Gentransfers. Die Schwierigkeiten
und die aktuellen Verbesserungen werden erörtert.
Abstract
Gene therapy raised euphoric expectations in the past that have yet to be met and
have even been lowered due to the absence of concrete clinical successes and the occurrence
of some tragic incidents. In spite of the great future potential of gene therapy,
numerous pilot studies in this field will probably be discontinued for a long time.
However, despite these failures, diseases will continue to be the aim of gene transfer
studies with experimental protocols using only temporary gene expression or those
restricted to individual organs and thus requiring only a low dose of the vehicle
(vector). The eye is exceptionally well suited as a potential target organ because
of its good and selective accessibility, low volume and the resultant low number of
gene ferries required as well as its special immunological status. The prognosis of
corneal grafting can be improved and profit from the methods catalogue of gene transfer
protocols. Moreover, the cornea can be used for ex vivo gene transfer before grafting.
Ophthalmology could thus occupy a pioneer position in clinical gene transfer. A survey
of the literature describes the current state of experimental improvement in corneal
grafting, the effect on scarring, neovascularization, and herpetic corneal infection.
The essential problem of gene therapy is the unsatisfactory gene transfer technique.
Difficulties and current improvements are discussed.
Schlüsselwörter
Gentransfer - Vektoren - Hornhauttransplantation - Narbenbildung - Neovaskularisation
- Herpesinfektion
Key words
Gene transfer - vectors - corneal grafting - scarring - neovascularization - herpes
infection
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Prof. Dr. Friedrich Hoffmann
Freie Universität Berlin · Universitätsklinikum Benjamin Franklin · Augenklinik und
Poliklinik
Hindenburgdamm 30
12200 Berlin
Email: fhoffman@zedat.fu-berlin.de
