Adeno-associated virus-mediated delivery of gdnf to skeletal...

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A61K 48/00 (2006.01) C07K 14/475 (2006.01) C12N 15/864 (2006.01)

Patent

CA 2469658

Compositions and methods for delivering GDNF to skeletal muscles to result in a therapeutic effect are disclosed. The compositions and methods use adeno- associated virus (AAV)-based gene delivery systems. The methods are useful for treating motoneuron diseases, such as amyotrophic lateral sclerosis (ALS).

Selon cette invention, des compositions et des méthodes sont utilisées pour administrer un facteur neurotrophique dérivé des cellules gliales (GDNF) dans des muscles du squelette afin qu'un effet thérapeutique soit obtenu. Ces compositions et méthodes font appel à des systèmes d'administration d'un gène fondés sur le virus adéno-associé (AAV). Ces méthodes permettent de traiter les maladies motoneuronales telles que la sclérose latérale amyotrophique (SLA).

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