IP Library Granted Patent US 8,912,153
Granted Patent B2
US 8,912,153 · App. 13/058,346 · Granted Dec 16, 2014

HDAC4 nucleic acid administration to treat retinal disease

Inventors: Constance L. Cepko (Newton, MA); Bo Chen (Cambridge, MA)
Assignee: President and Fellows of Harvard College
C07K14/4702A01K67/027C12N9/16C12N15/63C12N15/85C12N15/67A61K2039/53A01K2207/05A01K2227/105A01K2267/03A61K48/00C12N2799/025
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Quick Facts
Patent No.
US 8,912,153
App. No.
13/058,346
Granted
Dec 16, 2014
Kind
B2
Abstract

Methods for inhibiting retinal cell death by altering expression of one or more of HDAC4, HDAC5, HDAC6, HDAC7, and HIF1α in a retinal cell are provided.

Claims (20)

1. A method of inhibiting death of a retinal cell compromised by a neurodegenerative eye disorder, comprising administering to the retina of a subject having the neurodegenerative eye disorder an isolated HDAC4 nucleic acid molecule, thereby inhibiting death of the retinal cell.

2. A method for treating or preventing a neurodegenerative eye disorder in a subject, comprising administering to the retina of said subject an isolated HDAC4 nucleic acid molecule, thereby treating or preventing said neurodegenerative eye disorder in said subject.

3. A method of inhibiting death of an isolated retinal cell compromised by a neurodegenerative eye disorder, comprising contacting said isolated retinal cell with an isolated HDAC4 nucleic acid molecule, thereby inhibiting death of the isolated retinal cell.

4. The method of claim 1 , wherein the HDAC4 nucleic acid molecule comprises a wild-type HDAC4 gene.

5. The method of claim 1 , wherein the retinal cell is a photoreceptor cell.

6. The method of claim 5 , wherein the photoreceptor cell is a rod cell.

7. The method of claim 5 , wherein the photoreceptor cell is a cone cell.

8. The method of any one of claim 1 , 2 , or 3 , wherein the retinal cell is selected from the group consisting of a bipolar cell, a rod photoreceptor cell and a cone photoreceptor cell.

9. The method of any one of claim 1 , 2 , or 3 , wherein the nucleic acid molecule is expressed in the cytoplasm.

10. The method of claim 9 , wherein the nucleic acid molecule comprises an HDAC4 gene comprising a mutation that causes the exogenous HDAC4 to be localized to the cytoplasm of the cell.

11. The method of claim 10 , wherein the HDAC4 is HDAC4-L175A.

12. The method of claim 10 , wherein the HDAC4 is HDAC4-4118.

13. The method of any one of claim 1 , 2 , or 3 , wherein the neurodegenerative eye disorder is age-related macular degeneration.

14. The method of any one of claim 1 , 2 , or 3 , wherein the neurodegenerative eye disorder is retinitis pigmentosa.

15. The method of any one of claim 1 , 2 , or 3 , wherein the nucleic acid molecule is contained within a vector.

16. The method of claim 15 , wherein the vector is selected from the group consisting of a retrovirus vector, an adenovirus vector, an adenoviral/retroviral chimera vector, an adeno-associated virus (AAV) vector, a herpes simplex virus I or II vector, a parvovirus vector, a reticuloendotheliosis virus vector, a poliovirus vector, a papillomavirus vector, a vaccinia virus vector and a lentivirus vector.

17. The method of claim 16 , wherein the vector is an AAV vector.

18. The method of claim 17 , wherein the AAV vector is an AAV 2/5 or an AAV 2/8 vector.

19. A method of inhibiting death of a bipolar cell in a subject, comprising administering to the retina of said subject an isolated HDAC6 nucleic acid molecule, thereby inhibiting death of said bipolar cell.

20. A method of inhibiting death of an isolated bipolar cell, comprising contacting said isolated bipolar cell with an isolated HDAC6 nucleic acid molecule, thereby inhibiting death of said bipolar cell.

Assignments (4)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 2, 2012
From: CEPKO, CONSTANCE L.; CHEN, BO
To: HOWARD HUGHES MEDICAL INSTITUTE
Reel/Frame 029059/0678 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 2, 2012
From: HOWARD HUGHES MEDICAL INSTITUTE
To: PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Reel/Frame 029059/0783 →
CONFIRMATORY LICENSE Recorded Aug 16, 2012
From: HARVARD UNIVERSITY, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 028795/0722 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 11, 2011
From: CEPKO, CONSTANCE L.; CHEN, BO
To: PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Reel/Frame 025942/0215 →
Continuity (2)
Provisional Application 61088455 · Aug 13, 2008
Related Publication 20110268705A1 · Nov 3, 2011