Zudem gelang es dem Team, das Überlesen der Mutation in Augen von Mäusen in vivo nachzuweisen.
"Unsere genbasierenden Therapiestrategien, die Genreparatur sowie die 'Read-through'-Therapie, stellen aus heutiger Sicht wertvolle, vielversprechende Alternativen zur Genaddition mittels Viren dar und dürften v.a. bei den großen und isoformreichen USH-Genen die einzigen Therapieoptionen sein.
www.uni-mainz.deThe team also successfully documented read-through of the mutation in vivo a mouse model.
"Our gene-based treatment strategies, involving gene repair as well as read-through therapy, represent valuable and promising alternatives to viral gene addition and may actually be the only treatment option for the large and isoform-rich USH genes.
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