Granted Patent
B2
US 12,201,698 · App. 17/383,052 · Granted Jan 21, 2025
Gene therapy for retinitis pigmentosa
Assignee:
GENZYME CORPORATION
A61K48/0058A61K9/0019A61K9/0048A61K31/7105A61K38/1709C12N15/113C12N15/86C12N2310/141C12N2320/34C12N2330/51C12N2750/14143
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Abstract
Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.
Claims (16)
1. A nucleic acid comprising an intron derived from an MVM comprising SEQ ID NO:23 and a promoter.
2. The nucleic acid of claim 1 , wherein the promoter is located 5′ to the MVM intron.
3. An expression construct comprising the nucleic acid of claim 1 .
4. A vector comprising the nucleic acid of claim 1 .
5. A cell comprising the nucleic acid of claim 1 .
6. The nucleic acid of claim 1 , further comprising an enhancer.
7. The nucleic acid of claim 2 , further comprising an enhancer.
8. An expression construct comprising the nucleic acid of claim 2 .
9. An expression construct comprising the nucleic acid of claim 7 .
10. A vector comprising the expression construct of claim 3 .
11. A vector comprising the expression construct of claim 8 .
12. A vector comprising the expression construct of claim 9 .
13. A cell comprising the expression construct of claim 3 .
14. A cell comprising the expression construct of claim 9 .
15. A cell comprising the vector of claim 4 .
16. A cell comprising the vector of claim 12 .
Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Aug 5, 2021
From: O'RIORDAN, CATHERINE; ADAMOWICZ, MATTHEW
To: GENZYME CORPORATION
Reel/Frame 057090/0545 →
Continuity (4)
Division
15127757
Provisional Application
61969027
· Mar 21, 2014
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