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Virale und nichtvirale Gentherapieansätze zur Behandlung von Netzhauterkrankungen

Viral and nonviral gene therapy for treatment of retinal diseases

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Zusammenfassung

Für die Behandlung von Netzhauterkrankungen eröffnet der Einsatz der Gentherapie neue Perspektiven. Die Verwendung von verschiedenartigen Oligonukleotiden oder viralen Expressionsvektoren erlaubt die Entwicklung von neuen Heilungsstrategien für Neovaskularisierungskrankheiten und retinale Degeneration. Therapeutische Oligonukleotide („Antisense“-Oligonukleotide, Aptamere und siRNA) können den gezielten Abbau von Transkripten und damit die Konzentrationsabnahme eines an der Pathogenese beteiligten Proteins induzieren. Dagegen wird mit viralen Vektoren (rAAV und Lentivirus) häufig die Funktion eines defekten Gens durch die eines gesunden ersetzt und so die Ursache der Krankheit bekämpft. Die an Tiermodellen erfolgreich angewandten Gentherapien führten bereits zur Entwicklung von Medikamenten, und weitere werden zurzeit klinisch erprobt.

Abstract

The development of gene therapeutic approaches offers new perspectives for the treatment of retinal diseases. The use of both, nonviral methods employing oligonucleotides as well as viral expression vectors provide the possibility to treat neovascularization defects and retinal degeneration, respectively. The mechanism by which the therapeutic oligonucleotides (antisense oligonucleotides, aptamers and siRNA) work is based on degradation of specific transcripts. Consequently, a reduction of the corresponding protein, which is involved in the particular pathogenesis, follows. In contrast, viral vector transduction can substitute the disease-associated gene with an intact copy. So far, animal models have successfully contributed to the development of gene therapeutic medication and further treatments are at the recruiting phase of clinical trials.

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  1. Abteilung für Medizinische Molekulargenetik und Gendiagnostik des Instituts für Medizinische Genetik, Universität Zürich, Schwerzenbach, Schweiz

    J. Neidhardt, K. Wycisk &  B. Klöckener-Gruissem

  2. Abteilung für Medizinische Molekulargenetik und Gendiagnostik des Instituts für Medizinische Genetik, Universität Zürich, Schorenstraße 16, 8603 , Schwerzenbach, Schweiz

    B. Klöckener-Gruissem

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  1. J. Neidhardt
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  2. K. Wycisk
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  3. B. Klöckener-Gruissem
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Correspondence to B. Klöckener-Gruissem.

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Neidhardt, J., Wycisk, K. & Klöckener-Gruissem, B. Virale und nichtvirale Gentherapieansätze zur Behandlung von Netzhauterkrankungen. Ophthalmologe 102, 764–771 (2005). https://doi.org/10.1007/s00347-005-1245-z

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  • DOI: https://doi.org/10.1007/s00347-005-1245-z

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