Methods and compositions for treating neuropathies

• US 8,551,945 B2
• Filed: 02/04/2010
• Issued: 10/08/2013
• Est. Priority Date: 02/04/2009
• Status: Active Grant

• Current Assignee: Sangamo Biosciences, Inc.
• Original Assignee: Sangamo Biosciences, Inc.
• Inventors: Dent, Carolyn, Laganiere, Josee, Meng, Xiangdong, Paschon, David, Tan, Siyuan, Zhang, Lei, Zhang, Steve H.
• Primary Examiner(s): Vivlemore, Tracy
• Assistant Examiner(s): Alley, Genevieve S
• Application Number:
  US12/658,258
• Publication Number:
  US 20110082078A1
• Time in Patent Office: 1,342 Days
• Field of Search: 514/6.9, 514/18.2, 435/325, 530/400, 536/23.5
• US Class Current: 514/6.9
• CPC Class Codes: A61K 38/00 Medicinal preparations containing peptides peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00
• CPC Class Codes: A61P 25/00 Drugs for disorders of the nervous system
• CPC Class Codes: A61P 25/02 for peripheral neuropathies
• CPC Class Codes: A61P 25/04 Centrally acting analgesics, e.g. opioids
• CPC Class Codes: A61P 25/16 Anti-Parkinson drugs
• CPC Class Codes: A61P 27/02 Ophthalmic agents
• CPC Class Codes: A61P 3/10 for hyperglycaemia, e.g. antidiabetics
• CPC Class Codes: A61P 7/12 Antidiuretics, e.g. drugs for diabetes insipidus ADH A61P5/10
• CPC Class Codes: C07K 14/4702 Regulators; Modulating activity

Abstract

Disclosed herein are methods and compositions for treating neuropathies by modulating endogenous NT-3 of GDNF gene expression.

Claims

1. An engineered zinc finger protein that modulates expression of NT-3 or GDNF, wherein the engineered zinc finger protein comprises six recognition helix regions ordered F1 to F6 and wherein the zinc finger protein is selected from the group consisting of a protein comprising the following amino acid sequences in the recognition helix regions:

2. A fusion protein comprising a zinc finger protein according to claim 1 and a functional domain.

3. The fusion protein of claim 2, wherein the functional domain is a transcriptional regulatory domain.

4. The fusion protein of claim 3, wherein the transcriptional regulatory domain is an activation domain.

5. The fusion protein of claim 3, wherein the transcriptional regulatory domain is a repression domain.

6. A polynucleotide encoding a zinc finger protein according to claim 1.

7. A pharmaceutical composition comprising a zinc finger protein according to claim 1.

8. A pharmaceutical composition comprising a polynucleotide according to claim 6.

9. An isolated cell comprising a zinc finger protein according to claim 1.

10. The isolated cell of claim 9, wherein the cell is a stem cell.

11. An isolated cell comprising a polynucleotide according to claim 6.

12. The isolated cell of claim 11, wherein the cell is a stem cell.

13. A method of activating NT-3 or GDNF in a cell, the method comprising introducing the zinc finger protein of claim 1 in a cell in vitro.

14. The method of claim 13, wherein the zinc finger protein is administered as a polynucleotide encoding the zinc finger protein.

15. The method of claim 14, wherein the polynucleotide is carried by a viral vector.

16. The method of claim 14, wherein the polynucleotide is carried by a non-viral vector.