IP Library › Granted Patent US 9,290,759
Granted Patent B2
US 9,290,759 · App. 13/818,956 · Granted Mar 22, 2016

Optimized miRNA constructs

Inventors: Asa Abeliovich (New York, NY); Hervé Rhinn (New York, NY); Toru Yamashita (New York, NY)
Assignee: THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
C12N15/113C12N15/111C12N2310/141C12N2330/00
View Patent ↗
Loading inventors, assignments & file history…
Monitor This Case
Get email alerts when status or documents change.
Order Certified Copies
Most orders are placed with the USPTO same day — all within 24 business hours.
Order via The Patent Place →
Pre-filled with this patent's details
Quick Facts
Patent No.
US 9,290,759
App. No.
13/818,956
Granted
Mar 22, 2016
Kind
B2
Abstract

The invention provides optimized miRNA sequences and their therapeutic use. The invention provides optimized miRNA constructs for treatment of neurodegenerative diseases.

Claims (8)

1. An engineered mammalian pre-microRNA comprising SEQ ID NO: 6, 8, 12, 14, 16, 18, 24, 26, 33, 34, 40, 42, 48, or 50.

2. A therapeutic composition for intracerebral delivery comprising a pre-microRNA comprising SEQ ID NOs: 6, 8, 12, 14, 16, 18, 24, 26, 33, 34, 40, 42, 48, or 50, or any combination thereof.

3. A method to reduce aSyn levels in a cell or in a subject in need thereof comprising administering to a cell or a subject in need thereof a therapeutic amount of a pre-micro RNA smiR-7-2(SNCA) comprising SEQ ID NO: 40 or 42, a pre-micro RNA smiR-153-1(SNCA) comprising SEQ ID NO: 6 or 8, a pre-micro RNA smiR-495(SNCA) comprising SEQ ID NO: 48 or 50, or any combination thereof.

4. A method to treat Parkinson's Disease in a subject in need thereof comprising administering to a subject in need thereof a therapeutic amount of a pre-micro RNA smiR-7-2(SNCA) comprising SEQ ID NO: 40 or 42, a pre-micro RNA smiR-153-1(SNCA) comprising SEQ ID NO: 6 or 8, a pre-micro RNA smiR-495(SNCA) comprising SEQ ID NO: 48 or 50, or any combination thereof.

5. A method to rescue or increase survival of dopaminergic neurons in a subject in need thereof comprising administering to a subject in need thereof a therapeutic amount of a pre-micro RNA smiR-7-2(SNCA) comprising SEQ ID NO: 40 or 42, a pre-micro RNA smiR-153-1(SNCA) comprising SEQ ID NO: 6 or 8, a pre-micro RNA smiR-495(SNCA) comprising SEQ ID NO: 48 or 50, or any combination thereof.

6. A method to rescue or increase survival of a dopaminergic neuron comprising contacting the neuron with a therapeutic amount of a pre-micro RNA smiR-7-2(SNCA) comprising SEQ ID NO: 40 or 42, a pre-micro RNA smiR-153-1(SNCA) comprising SEQ ID NO: 6 or 8, a pre-micro RNA smiR-495(SNCA) comprising SEQ ID NO: 48 or 50, or any combination thereof.

7. A method to reduce APP levels in a cell or in a subject in need thereof comprising administering to a cell or a subject in need thereof a therapeutic amount of a pre-micro RNA smiR-106(a)(APP) comprising SEQ ID NO: 26, a pre-micro RNA smiR-153(APP) comprising SEQ ID NO: 18, a smiR-101-1(APP) comprising SEQ ID NO: 14, or any combination thereof.

8. A method to treat Alzheimer's disease in a subject in need thereof comprising administering to a subject in need thereof a therapeutic amount of a pre-micro RNA smiR-106(a)(APP) comprising SEQ ID NO: 26, a pre-micro RNA smiR-153(APP) comprising SEQ ID NO: 18, a smiR-101-1(APP) comprising SEQ ID NO: 14, or any combination thereof.

Assignments (2)
CONFIRMATORY LICENSE Recorded Jun 27, 2016
From: COLUMBIA UNIV NEW YORK MORNINGSIDE
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 039170/0616 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 19, 2013
From: ABELIOVICH, ASA; YAMASHITA, TORU; RHINN, HERVE
To: THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
Reel/Frame 030837/0294 →
Continuity (2)
Provisional Application 61376922 · Aug 25, 2010
Related Publication 20140051748A1 · Feb 20, 2014