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US 8,007,814 B2Granted
Therapy for lysosomal enzyme deficiencies
AssigneeUniversity of Massachusetts
InventorsEdward I. Ginns, Gary R. Ostroff
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Abstract
The present invention provides a therapeutic delivery system comprising an extracted yeast cell wall comprising beta-glucan, a payload trapping molecule and a payload molecule, wherein the payload molecule and the payload trapping molecule are soluble in the same solvent system wherein the payload molecule supplements the function of the deficient lysosomal enzyme. The invention further provides methods of making and methods of using the therapeutic delivery system.
Claims31
A therapeutic delivery system comprising:
- an extracted yeast cell wall defining an internal space and comprising about 6 to about 90 weight percent beta-glucan;
- a payload trapping molecule and a payload molecule selected from the group consisting of a nucleic acid, a peptide, a protein and a mixture thereof in an amount effective to supplement the function of a deficient lysosomal enzyme, wherein the deficient lysosomal enzyme results in an affected lysosomal function, and wherein the affected lysosomal function is a defective degradation of sphingolipid components related to a lysosomal storage disorder selected from the group consisting of Gaucher disease type 1, Gaucher disease type 2 and Gaucher disease type 3;
- wherein the payload molecule and the payload trapping molecule are soluble in the same solvent system;
- wherein the payload trapping molecule is contained within the internal space defined by the extracted yeast cell wall;
- and wherein the payload trapping molecule is present in an amount sufficient to facilitate retention of the payload molecule within the extracted yeast cell wall.
2 more elements
A method of making a therapeutic delivery system comprising the steps of:
- providing an extracted yeast cell wall defining an internal space and comprising about 6 to about 90 weight percent beta-glucan;
- contacting the extracted yeast cell wall with a payload molecule selected from the group consisting of a nucleic acid, a peptide, a protein and a mixture thereof in an amount effective to supplement the function of a deficient lysosomal enzyme, wherein the deficient lysosomal enzyme results in an affected lysosomal function, and wherein the affected lysosomal function is a defective degradation of sphingolipid components related to a lysosomal storage disorder selected from the group consisting of Gaucher disease type 1, Gaucher disease type 2 and Gaucher disease type 3;
- contacting the extracted yeast cell wall with a payload trapping molecule wherein the payload molecule and the payload trapping molecule are soluble in the same solvent system;
- wherein the payload trapping molecule is contained within the internal space defined by the extracted yeast cell wall;
- and wherein the payload trapping molecule is present in an amount sufficient to facilitate retention of the payload molecule within the extracted yeast cell wall to form the therapeutic delivery system.
2 more elements
Dependencies are inferred from the claim text.