Método para mejorar la integridad deteriorada de la unión neuromuscular que comprende la administración de una composición que comprende un vector viral adeno-asociado recombinante (raav) que comprende una molécula de ácido nucleico que codifica la alfa-glucosidasa ácida (gaa).

摘要

The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.