Therapeutische Ansätze bei Patienten mit Retinitis pigmentosa

Zusammenfassung

Hintergrund: Retinitis pigmentosa (RP) bezeichnet einen genetisch und klinisch heterogenen Formenkreis an dystrophischen Netzhauterkrankungen. Im Verlauf der Erkrankung kommt es zu zunehmenden Gesichtsfeldeinschränkungen bis hin zur Erblindung. Bisher ist keine Therapie etabliert. Durch zunehmendes Wissen über die zugrunde liegenden genetischen und pathophysiologischen Veränderungen gibt es eine Reihe von neuen Therapieansätzen, von denen einige hier vorgestellt werden sollen. Methodik: Es wurde eine systematische Literaturrecherche in PubMed zu definierten Stichworten durchgeführt. Ergebnisse: Zu den neuen Therapieansätzen gehören Gentherapie, pharmakologische Substanzen, Neuroprotektion, Elektrostimulation, retinale Implantate, Zelltransplantation und optogenetische Ansätze. Schlussfolgerung: In den letzten Jahren gab es einige Fortschritte in der Erforschung möglicher Therapieansätze bei dystrophischen Netzhauterkrankungen. Die Forschung ist in den einzelnen Bereichen unterschiedlich weit fortgeschritten. Obwohl es nach wie vor keine etablierte Therapie gibt, stehen die Chancen gut, dass in Zukunft zumindest einem Teil der RP-Patienten eine Therapie angeboten werden kann.

Abstract

Background: Retinitis pigmentosa (RP) is a clinically and genetically heterogenous group of hereditary retinal disorders, which lead to progressive loss of vision and finally blindness. Yet there is no approved therapy. Advances in unravelling underlying genetic disorders and pathophysiological mechanisms offer new therapeutic approaches of which some are summarised in this review. Methods: We performed a systematic literature research for defined key words in PubMed. Results: New approaches to therapy for RP include: gene therapy, pharmacological treatment, neuroprotection, electrical stimulation, retinal prostheses, retinal transplantation and optogenetic therapy. Conclusions: Recently there have been advances in new approaches for therapy of dystrophic retinal diseases. Advances in the different approaches are being made at different rates. Although there is no approved therapy yet, the future for treating RP at least in some patients looks promising.

Schlüsselwörter

Retinitis pigmentosa - Therapie - Gentherapie - Neuroprotektion - Netzhautprothese

Key words

retinitis pigmentosa - therapy - gene therapy - neuroprotection - retinal prosthesis

• Literatur

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