IP Library › Granted Patent US 12,325,856
Granted Patent B2
US 12,325,856 · App. 17/374,147 · Granted Jun 10, 2025

Recombinant virus products and methods for inhibition of expression of myotilin

Inventors: Scott Quenton Harper (Powell, OH); Jian Liu (Savoy, IL)
Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
C12N15/113C07K14/4707C12N15/1138C12N15/86C12N2310/14C12N2310/141C12N2320/32C12N2330/51C12N2750/14143
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Quick Facts
Patent No.
US 12,325,856
App. No.
17/374,147
Granted
Jun 10, 2025
Kind
B2
Abstract

The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A.

Claims (10)

1. A recombinant adeno-associated virus encoding a myotilin (MYOT) microRNA (miRNA) comprising an antisense guide strand as set out in any one of SEQ ID NOs: 7701, 7798, 7945, 7967, 7990, 8057, 8078, and 8148, wherein the recombinant adeno-associated virus lacks rep and cap genes.

2. A composition comprising the recombinant adeno-associated virus of claim 1 and a carrier, diluent, and/or adjuvant.

3. The recombinant adeno-associated virus of claim 1 , wherein the recombinant adeno-associated virus (rAAV) is rAAV-6.

4. The recombinant adeno-associated virus of claim 3 , wherein the rAAV-6 is self-complementary rAAV-6.

5. A method of inhibiting expression of the myotilin (MYOT) gene in a cell comprising contacting the cell with an effective amount of the recombinant adeno-associated virus of claim 1 .

6. A method of delivering a myotilin (MYOT) miRNA-encoding DNA to an animal in need thereof, comprising administering to the animal an effective amount of the recombinant adeno-associated virus of claim 1 .

7. A method of treating limb girdle muscular dystrophy type 1A in a subject comprising administering to the subject an effective amount of the recombinant adeno-associated virus of claim 1 .

8. The method of claim 5 , wherein the recombinant adeno-associated virus is a self-complementary rAAV-6.

9. The method of claim 6 , wherein the recombinant adeno-associated virus is a self-complementary rAAV-6.

10. The method of claim 7 , wherein the recombinant adeno-associated virus is a self-complementary rAAV-6.

Assignments (4)
CORRECTIVE ASSIGNMENT TO CORRECT THE INCORRECT SERIAL NUMBER PCT/US14/47999 FILED 07/24/2014 SHOULD BE PCT/US12/47999 FILED 07/24/2012 PREVIOUSLY RECORDED ON REEL 063643 FRAME 0786. ASSIGNOR(S) HEREBY CONFIRMS THE THE CORRECT SERIAL NUMBER AND ASSIGNMENT DOC ID 507906702. Recorded May 24, 2023
From: NATIONWIDE CHILDREN'S HOSPITAL, INC.
To: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Reel/Frame 063743/0208 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 15, 2023
From: NATIONWIDE CHILDREN'S HOSPITAL, INC.
To: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Reel/Frame 063643/0786 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 17, 2021
From: HARPER, SCOTT QUENTON; LIU, JIAN
To: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Reel/Frame 058417/0728 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 17, 2021
From: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
To: NATIONWIDE CHILDREN'S HOSPITAL
Reel/Frame 058417/0958 →
Continuity (6)
Division 16144432 · Sep 27, 2018
Continuation 14853517 · Sep 14, 2015
Continuation In Part 14058909 · Oct 21, 2013
Continuation In Part PCTUS2012034408 · Apr 20, 2012
Provisional Application 61478012 · Apr 21, 2011
Related Publication 20220064641A1 · Mar 3, 2022
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