Title: | Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme |
Journal : | Molecular therapy, 6, 5 |
Authors: | Raben N ; Jatkar T ; Lee A ; Lu N ; Dwivedi S ; Nagaraju K ; Plotz PH |
Material Type: | Article |
Publication Date: | 2002 |
Size: | p. 601-608 |
Languages: | English |
Keywords : | acid alpha-glucosidase ; animal models ; enzymotherapy ; GAA gene ; heart muscle ; mouse model ; Pompe disease ; skeletal muscle ; transgenic animals |