Method for improving impaired Integrity of the neuromuscular Junction comprising Administration of a composition comprising a recombinant adeno-associated viral vector (rAAV) comprising a Nucleic Acid Molecule that encodes The Acid Alpha glucosidase (GAA).
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.uso de una composición que comprende un vector rAAV que comprende una secuencia de ácido nucleico pseudotipificada de repetición terminal de AAV2 con una proteína cápside AAV9(vector rAAV2/9) que sirve para preparar un medicamento útil para mejorar la integridad deteriorada de la unión neuromuscular, que comprende el uso en un sujeto con integridad deteriorada de la unión neuromuscular,.
