IP Library › Granted Patent US 10,301,649
Granted Patent B2
US 10,301,649 · App. 15/295,591 · Granted May 28, 2019

Recombinant virus products and methods for inhibition of expression of DUX4

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Quick Facts
Patent No.
US 10,301,649
App. No.
15/295,591
Granted
May 28, 2019
Kind
B2
Abstract

The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.

Claims (59)

1. A recombinant adeno-associated virus comprising:

a) the DUX4 miRNA-encoding DNA comprising the nucleotide sequence of SEQ ID NO: 1 or SEQ ID NO: 2, or

b) a DNA encoding a DUX4 miRNA comprising an miRNA antisense guide strand, wherein the miRNA antisense guide strand consists of the nucleotide sequence of

SEQ ID NO: 8482,

SEQ ID NO: 8372,

SEQ ID NO: 8371,

SEQ ID NO: 8370,

SEQ ID NO: 8367,

SEQ ID NO: 8366,

SEQ ID NO: 8365,

SEQ ID NO: 8219,

SEQ ID NO: 8218,

SEQ ID NO: 8152,

SEQ ID NO: 8147,

SEQ ID NO: 8145,

SEQ ID NO: 7397,

SEQ ID NO: 7396,

SEQ ID NO: 7395,

SEQ ID NO: 7108,

SEQ ID NO: 7107,

SEQ ID NO: 7106,

SEQ ID NO: 6633,

SEQ ID NO: 6631,

SEQ ID NO: 6622,

SEQ ID NO: 6619,

SEQ ID NO: 6609,

SEQ ID NO: 6608,

SEQ ID NO: 6568,

SEQ ID NO: 6561 or

SEQ ID NO: 6560,

wherein the recombinant adeno-associated virus lacks rep and cap genes.

2. A DNA encoding a DUX4 miRNA comprising an miRNA antisense guide strand, wherein the miRNA antisense guide strand consists of the nucleotide sequence of

SEQ ID NO: 8482,

SEQ ID NO: 8372,

SEQ ID NO: 8371,

SEQ ID NO: 8370,

SEQ ID NO: 8367,

SEQ ID NO: 8366,

SEQ ID NO: 8365,

SEQ ID NO: 8219,

SEQ ID NO: 8218,

SEQ ID NO: 8152,

SEQ ID NO: 8147,

SEQ ID NO: 8145,

SEQ ID NO: 7397,

SEQ ID NO: 7396,

SEQ ID NO: 7395,

SEQ ID NO: 7108,

SEQ ID NO: 7107,

SEQ ID NO: 7106,

SEQ ID NO: 6633,

SEQ ID NO: 6631,

SEQ ID NO: 6622,

SEQ ID NO: 6619,

SEQ ID NO: 6609,

SEQ ID NO: 6608,

SEQ ID NO: 6568,

SEQ ID NO: 6561 or

SEQ ID NO: 6560.

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 Apr 23, 2018
From: HARPER, SCOTT Q.; LIU, JIAN; COPPENS, SARA; WALLACE, LINDSAY
To: NATIONWIDE CHILDREN'S HOSPITAL, INC.
Reel/Frame 045614/0273 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 11, 2016
From: HARPER, SCOTT Q.; LIU, JIAN; COPPENS, SARA; WALLACE, LINDSAY
To: NATIONWIDE CHILDREN'S HOSPITAL, INC.
Reel/Frame 040283/0616 →
Cited By (2)
US 12,486,328 US 12,529,056