Gene therapy for LAMA2 MD / MDC1A

• Unité de recherche: INNOSUISSE
• Numéro de projet: 34870.1 IP-LS
• Titre du projet anglais: Gene therapy for LAMA2 MD / MDC1A

Description succincte (Allemand)

Gene therapy for LAMA2 MD / MDC1A

Description succincte (Anglais)

Gene therapy for LAMA2 MD / MDC1A

Résumé des résultats (Abstract) (Allemand)

Muscular dystrophies are untreatable, fatal diseases. Previous mouse experiments showed that two linker proteins prevent early lethality and stabilize muscle function. We now aim to deliver the linker proteins by AAV9 vectors to test whether this method can be used to treat human patients.