Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
AssigneeBioasis Technologies, Inc.
InventorsChristopher M. Starr, Todd Zankel
Life of this patent
Estimating the term…
Abstract
The present invention provides for compositions and methods for treating, ameliorating or preventing a lysosomal storage disease by administering to a patient suffering front a lysosomal storage disease a P97 conjugated with art enzyme which is capable of transportation into the lysosomes of cells on either sides of the blood brain barrier.
Claims6
A method for treating a subject having a lysosomal storage disease, said method comprising
- administering a pharmaceutical composition to the subject wherein the composition comprises a p97 molecule covalently linked to a protein whose deficiency causes the disease.
2-13. (canceled)
A compound comprising a p97 molecule covalently linked to a protein whose deficiency causes a lysosomal storage disease.
15-20. (canceled)
A method of screening a compound for therapeutic activity in treating a lysosomal storage disease, said method comprising:
- contacting a cell having a lysosome with the compound, wherein the compound comprises p97 covalently linked to a protein deficient in a lysosomal storage disease;
- and
- monitoring delivery of the compound to the lysosome.
22-29. (canceled)
Dependencies are inferred from the claim text.