IP Library › Granted Patent US 10,954,518
Granted Patent B2
US 10,954,518 · App. 16/295,621 · Granted Mar 23, 2021

RAAV-based compositions and methods for treating amyotrophic lateral sclerosis

Inventors: Christian Mueller (Concord, MA); Robert H. Brown, Jr. (Needham, MA)
Assignee: University of Massachusetts
C12N15/113C12N15/111C12N15/1137C12N15/86C12Y115/01001C12N2310/14C12N2310/141C12N2320/32C12N2330/51C12N2750/14143
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Quick Facts
Patent No.
US 10,954,518
App. No.
16/295,621
Granted
Mar 23, 2021
Kind
B2
Abstract

The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.

Claims (8)

1. A method of inhibiting expression of C9orf72 in a subject in need thereof, the method comprising administering to the subject an effective amount of a synthetic miRNA that targets a C9orf72 RNA transcript, wherein the synthetic miRNA specifically binds to a nucleic acid sequence of the RNA transcript, wherein the nucleic acid sequence of the RNA transcript bound by the synthetic miRNA is encoded by a nucleic acid sequence set forth in SEQ ID NO: 2, 6, or 7.

2. The method of claim 1 , wherein the synthetic miRNA further comprises flanking regions of miR-155.

3. The method of claim 2 , wherein the RNA transcript is a pre-mRNA transcript.

4. The method of claim 2 , wherein the RNA transcript comprises a G 4 C 2 hexanucleotide repeat.

5. The method of claim 3 , wherein the pre-mRNA transcript is a C9orf72 V 1 isoform transcript.

6. The method of claim 3 , wherein the pre-mRNA transcript is a C9orf72 V 3 isoform transcript.

7. The method of claim 3 , wherein the pre-mRNA transcript is not a C9orf72 V 2 isoform transcript.

8. The method of claim 1 , wherein the RNA transcript comprises an intron.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 20, 2019
From: MUELLER, CHRISTIAN; BROWN, ROBERT H., JR.
To: UNIVERSITY OF MASSACHUSETTS
Reel/Frame 051059/0941 →
Continuity (3)
Continuation 15126688
Provisional Application 61955189 · Mar 18, 2014
Related Publication 20200032256A1 · Jan 30, 2020
Cited By (3)
US 12,312,587 US 12,529,051 US 12,662,669