Visser, T.P.

lv-sf-gaa lv-sf-gfp lv-sf-gaa –treated mice wt mice gaa ϫ/ϫ mice disease transplantation vector transplant –treated signi ficantly glycogen level signi figure therapy pompe blood hematopoietic muscle erasmus mc tissue heart number ef ficacy gaa activity expression activity enzyme spleen mouse function pompe disease ficantly month storage enzyme replacement therapy treatment erasmu lv-sf-gfp mice muscle strength protein gene therapy liver gaa expression animal volume patient ficacy marrow acid ␣- glucosidase signi ficant difference performance bone marrow transplantation motor performance bm cells promoter reduction glycogen storage 8 months 10 months pressure hematopoietic cells lv-sf-gfp –treated mice difference ficant system motor 2010. brain analysis strength heart rate october glycogen deposition glycogen content wall thickness hsc transplantation glucosidase library