Tissue Engineering und Gentherapie des Bewegungsapparates mit Muskelzellen

Zusammenfassung.

Ziel: Der Einsatz von Muskelgewebe in der somatischen Gentherapie ermöglicht den Transfer von Genen in betimmte Gewebe mit einer biochemischen Funktionsstörung. Methode: Muskelgewebe ist im Einsatz der Gentherapie und im Tissue Engineering vielversprechend. Viele Muskelgruppen sind per injectionem direkt erreichbar und können wiederholt biopsiert und in relativ großen Mengen gewonnen werden, ohne die Gesundheit des Patienten zu beeinträchtigen. Muskelgewebe besteht aus vielkernigen, postmitotischen Muskelfasern, welche eine hohe und langfristige transgene Expression ermöglichen. Letztendlich ist Muskelgewebe gut vaskularisiert, was über die Blutbahn eine systemische Wirksamkeit erzielen lässt. Ergebnisse: Zellen muskulären Ursprungs können die Muskelheilung und Knochenheilung fördern. Transplantierte Zellen muskulären Ursprungs ermöglichen eine lang anhaltende Expression von therapeutischen Proteinen. Die aus der Muskulatur isolierten Stammzellen sind pluripotent und können u. a. in Osteoblasten differenzieren. Schlussfolgerung: Diese Charakeristika voraussetzend sind im Folgenden vier Anwendungsmöglichkeiten des Tissue Engineerings und der Gentherapie mit Muskelgewebe beschrieben: Erbliche Muskelerkrankungen, Muskelverletzungen und Rekonstruktion, Knochenheilung und intraartikuläre Läsionen.

Aim: Muscle-based somatic gene therapy is a novel way to alleviate a biochemical deficiency. Method: Muscle-derived cells are very promising in the field of gene therapy and tissue engineering. First, most muscle tissue is accessible by injection. Second, muscle tissue consists of multinucleated, postmitotic myofibers, which enable a long-term expression of the transduced gene. Third, muscle tissue can be biopsied easily. It is available in abundance and the biopsy does not compromise the health and function of the patient. Finally, muscle tissue is highly vascularized, which makes systemic delivery feasible. Results: Muscle-derived cells can promote muscle healing and bone healing. Implanted cells maintain a long-term transgene expression of therapeutic proteins. Isolated, muscle-derived stem cells can differentiate in osteoblasts. Conclusion: Based on these characteristics, we present four possible applications: inherited muscular diseases, muscle injury, bone healing, and intraarticular disorders.

Schlüsselwörter:

Gentherapie - Stammzellen - Muskelverletzung - Knochenheilung - Arthrose

Key words:

Gene therapy - stem cell - muscle injury - bone healing - arthritis

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