IP Library Granted Patent US 9,486,521
Granted Patent B2
US 9,486,521 · App. 14/087,206 · Granted Nov 8, 2016

Therapeutic applications targeting SARM1

Inventors: Marc Freeman (Barre, MA); Stephan Zuchner (Pinecrest, FL)
Assignees: University of Massachusetts; University of Miami
A61K39/3955A61K31/7088A61K31/713A61K38/00G01N33/5058G01N33/566G01N33/6896G01N2500/04G01N2800/2814G01N2800/2835
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Quick Facts
Patent No.
US 9,486,521
App. No.
14/087,206
Granted
Nov 8, 2016
Kind
B2
Abstract

The present disclosure provides methods for reducing axonal and/or synaptic degradation in neurons by modulating sterile α/Armadillo/Toll-Interleukin receptor homology domain protein (SARM) activity and/or expression.

Claims (14)

1. A method for reducing axonal or synaptic degradation in a neuron, the method comprising:

selecting a neuron with or at an increased risk for developing axonal or synaptic degradation; and

contacting the neuron with an effective amount of a composition comprising a small interfering RNA (siRNA) that binds to sterile α/Armadillo/Toll-Interleukin receptor homology domain protein (SARM) mRNA for a time sufficient to inhibit SARM expression, thereby reducing axonal or synaptic degradation in the neuron.

2. The method of claim 1 , wherein the siRNA binds to SARM mRNA comprising a nucleic acid sequence selected from the group consisting of SEQ ID NO: 1, 3 and 5.

3. A method for reducing axonal or synaptic degradation in a subject with or at risk for developing axonal or synaptic degradation, the method comprising:

selecting a subject with or at an increased risk for developing axonal or synaptic degradation; and

treating the subject with an effective amount of a composition comprising a siRNA compound that binds to SARM mRNA for a time sufficient to inhibit SARM expression, thereby reducing axonal or synaptic degradation in the subject.

4. The method of claim 3 , wherein the subject at an increased risk for developing axonal or synaptic degradation is a subject that has experienced trauma of the CNS or PNS but that does not present symptoms of neurodegenerative disease.

5. The method of claim 3 , wherein the subject at an increased risk for developing axonal or synaptic degradation is a subject with diabetes but without diabetic neuropathy.

6. The method of claim 3 , wherein the subject at an increased risk for developing axonal or synaptic degradation is a subject scheduled to be exposed to a chemotherapeutic agent associated with the onset and/or development of neurodegeneration.

7. The method of claim 3 , wherein the subject at an increased risk for developing axonal or synaptic degradation is a subject scheduled to undergo or undergoing chemotherapy, or treatment with a toxin associated with neurodegeneration.

8. The method of claim 3 , wherein the subject has or is at an increased risk of neurodegenerative disease.

9. The method of claim 3 or 8 , wherein the axonal or synaptic degradation is in the central nervous system (CNS) or the peripheral nervous system (PNS).

10. The method of claim 9 , wherein the axonal or synaptic degradation is in the PNS.

Assignments (3)
CONFIRMATORY LICENSE Recorded Dec 1, 2017
From: UNIVERSITY OF MASSACHUSETTS MEDICAL SCH
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 044593/0583 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 2, 2014
From: FREEMAN, MARC
To: UNIVERSITY OF MASSACHUSETTS
Reel/Frame 031873/0068 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 2, 2014
From: ZUCHNER, STEPHAN
To: UNIVERSITY OF MIAMI
Reel/Frame 031873/0071 →
Continuity (3)
Division 13530998 · Jun 22, 2012
Provisional Application 61501111 · Jun 24, 2011
Related Publication 20140079712A1 · Mar 20, 2014