| abstract |
The present invention treats lysosomal storage diseases by administering p97 conjugated to an enzyme that can be transported to the lysosomes of cells on either side of the blood brain barrier to patients suffering from lysosomal storage diseases. Provides compositions and methods for amelioration or prevention. In one embodiment, the method of the invention is a method for treating a subject having lysosomal storage disease, the method comprising administering to the subject a pharmaceutical composition, wherein Thus, the composition comprises a p97 molecule covalently linked to a protein that, when deficient, causes the disease. |