Genomeditierung in der Zell- und Gentherapie

Zusammenfassung

Gen- und zelltherapeutische Behandlungsansätze dienen als vielversprechende Grundlage zur Entwicklung neuer Therapieoptionen bei erworbenen und angeborenen Erkrankungen, für die bislang keine kurativen Behandlungsmethoden zur Verfügung stehen. Mit der Etablierung humaner induzierter pluripotenter Stammzellen vor 10 Jahren sowie der rasanten Entwicklung der Genomeditierung mittels Designernukleasen, insbesondere des CRISPR/Cas9-Systems, bieten sich der klinischen Medizin vielfältige Therapieoptionen an, die sich in noch nie dagewesener Geschwindigkeit weiterentwickeln und im Begriff stehen, klinische Routine verschiedenster Fachrichtungen auf revolutionäre Weise zu verändern. Auch für die Transfusionsmedizin, als klassisch zelltherapeutischem Fachgebiet, bedeuten diese Technologieplattformen ein weites Spektrum an Entwicklungsmöglichkeiten. Mit diesem Übersichtsartikel fassen wir die wesentlichen Grundlagen und den aktuellen Stand der Forschung, einschließlich experimenteller klinischer Studien, zusammen und bieten einen Ausblick auf mögliche Szenarien der Zukunft für die Stammzellforschung und die Gentherapie.

Abstract

Novel gene and cell therapeutic paradigms have opened promising avenues for treating acquired as well as hereditary disease entities that currently lack viable treatment options. The introduction of human induced pluripotent stem cells a decade ago in conjunction with the rapid development of genome editing approaches, most notably CRISPR-Cas9, offer unprecedented therapeutic opportunities. Transfusion medicine, with its traditional strong standing in cell therapy, can contribute to shaping the imminent clinical change and the impact these fields are about to have. In this review, we summarize fundamental principles, current research and clinical studies, and provide a perspective of future biomedical realities originating from these developments in stem cell research and gene therapy.

Schlüsselwörter

Gentherapie - Zelltherapie - Genomeditierung - personalisierte Medizin - Stammzellen - CRISPR - TALEN - ZFN

Key words

gene therapy - cell therapy - genome editing - personalized medicine - stem cells - CRISPR - TALEN - ZFN

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