Einführung in die Gentherapie

Zusammenfassung

Nach vielen Jahren der Entwicklung und zahlreichen Rückschlägen hat die Gentherapie, also die direkte therapeutische Modifikation der Gene auf DNA- oder RNA-Ebene, erste unbestreitbare Erfolge. Dabei werden sowohl „ex vivo“ Verfahren, also die genetische Manipulation von zuvor entnommenen Körperzellen, als auch „in vivo“ Methoden erfolgreich eingesetzt. Neben dem Ersatz fehlender oder schadhafter Gene können auch die Korrektur der DNA-Sequenz durch die CRISPR-Cas9-„Genschere“ und die sequenzspezifische Beeinflussung der Genexpression auf RNA-Ebene als gentherapeutische Methoden im weiteren Sinne bezeichnet werden.

Abstract

After many years of research and numerous setbacks, there are now undeniable success stories of gene therapies, namely the direct modification of genetic information on the DNA or RNA level. Both “ex vivo” strategies, i. e. the genetic manipulation of patient cells in a dish, as well as “in vivo” approaches are being used successfully. In addition to the supplementation of defective genes, the use of the CRISPR-Cas9 system to alter nuclear DNA sequences and the sequence-specific interference with the transcriptional process on the RNA level can be designated as gene therapies in a broad sense.

Schlüsselwörter

Gentherapie - Virale Vektoren - Antisense-Oligonukleotide - siRNA

Key words

gene therapy - viral vectors - antisense oligonucleotides - siRNA

Publikationsverlauf

Eingereicht: 14. September 2022

Angenommen: 12. Dezember 2022

Artikel online veröffentlicht:
20. Februar 2023

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