Autophagy and phospholipidosis pathway assays
First Claim
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1. A method of detecting the presence of a lysosomal storage disease in a subject comprising the steps of:
- (a) obtaining a sample containing cells from said subject;
(b) contacting said sample with a cationic amphiphilic tracer compound that localizes to a vacuole in a cell selected from
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Abstract
Provided are assays useful for detecting and monitoring autophagy and phospholipidosis, including the progression of lysosomal storage diseases. Drugs and treatments for lysosomal storage diseases can be monitored for effectiveness in lysosomal storage disease conditions. Drug candidates and suspected toxic agents can also be screened for toxicity to cells, tissues and organs. Also provided are methods for distinguishing between phospholipidosis activators and autophagy pathway perturbation agents.
57 Citations
17 Claims
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1. A method of detecting the presence of a lysosomal storage disease in a subject comprising the steps of:
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(a) obtaining a sample containing cells from said subject; (b) contacting said sample with a cationic amphiphilic tracer compound that localizes to a vacuole in a cell selected from - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13)
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14. A method of detecting Fabry disease, phospholipodosis, or lysosomal perturbation in U2OS human cells comprising the steps of:
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(a) obtaining a sample containing said cells; (b) contacting said sample with the cationic amphiphilic tracer compound having the structure - View Dependent Claims (16)
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15. A method of detecting lysosomal perturbation in HeLa cells comprising the steps of:
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(a) obtaining a sample containing said cells; (b) contacting said sample with a cationic amphiphilic tracer compound selected from
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17. A method of detecting Niemann-Pick disease in human cells comprising the steps of:
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(a) obtaining a sample containing said cells; (b) contacting said sample with the cationic amphiphilic tracer compound having the structure
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Specification