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US 10,874,750 B2Granted
Gene therapy constructs and methods of use
AssigneeAmicus Therapeutics, Inc.
InventorsHung Do, Steven Tuske, Russell Gotschall, Ce Feng Liu
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Abstract
Provided herein are improved gene therapy vectors and methods of use, in some embodiments, comprising sequences for improved expression and cellular targeting of a therapeutic protein.
Claims19
A gene therapy vector comprising a nucleic acid construct encoding a polypeptide comprising:
- (a) a therapeutic protein;
- (b) a peptide that binds to the cation-independent mannose 6-phosphate (M6P) receptor (CI-MPR), wherein the peptide is a variant (vIGF2) peptide comprising the sequence of SEQ ID NO:
- 31;
- (c) a linker between the therapeutic protein and the peptide that binds CI-MPR;
- and
- (d) a signal peptide selected from a binding immunoglobulin protein (BiP) signal peptide and a Gaussia signal peptide.
3 more elements
Dependencies are inferred from the claim text.