Therapeutic and diagnostic methods and compositions for neurodegenerative diseases
AssigneeWisconsin Alumni Research Foundation
InventorsSu-Chun Zhang, Hong Chen
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Abstract
Methods and compositions relating to motor neurons derived from induced pluripotent stem cells of subjects having a neurodegenerative disease, where the motor neurons exhibit phenotypes characteristic of the neurodegenerative disease, are provided herein. In particular, the present invention provides methods for screening putative therapeutic agents and methods for diagnosing living subjects as having a neurodegenerative disease. In addition, the present invention provides therapeutic gene transfer methods for treating or preventing a neurodegenerative disease in a subject in need thereof.
Claims16
A method of evaluating a test compound for a neuroprotective effect, the method comprising the steps of:
- (i) contacting a test compound to motor neurons;
- and
- (ii) measuring a level of neurofilament light (NF-L) polypeptide or NF-L mRNA in the contacted motor neurons, wherein an increased level in NF-L polypeptide or NF-L mRNA relative to motor neurons that have not been exposed to the test compound indicates that the compound provides a neuroprotective effect.
A method for identifying a therapeutic agent to treat a neurodegenerative disease, the method comprising the steps of:
- (i) contacting a test compound to motor neurons;
- and
- (ii) measuring a level of neurofilament light (NF-L) polypeptide or NF-L mRNA in the contacted motor neurons, wherein an increased level in NF-L polypeptide or NF-L mRNA relative to motor neurons that have not been exposed to the therapeutic agent indicates that the agent treats a neurodegenerative disease.
A method for identifying a therapeutic agent to treat a neurodegenerative disease
- (i) contacting a test compound to motor neurons;
- and
- (ii) measuring the level of phosphorylated neurofilament in the contacted motor neurons, wherein a decrease in the level of phosphorylated neurofilament relative to motor neurons that have not been exposed to the therapeutic agent indicates that the agent treats a neurodegenerative disease.
Dependencies are inferred from the claim text.