IP Library Granted Patent US 9,944,910
Granted Patent B2
US 9,944,910 · App. 15/075,457 · Granted Apr 17, 2018

TATk-CDKL5 fusion proteins, compositions, formulations, and use thereof

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Quick Facts
Patent No.
US 9,944,910
App. No.
15/075,457
Granted
Apr 17, 2018
Kind
B2
Abstract

Disclosed herein are compositions and formulations containing a TATk-CDKL5 fusion protein. Also disclosed are methods of producing a TATk-CDKL5 fusion protein from vectors containing a TATk-CDKL5 cDNA and methods of transducing cells with the vectors containing a TATk-CDKL5 cDNA and the TATk-CDKL5 fusion protein.

Claims (47)

1. A fusion protein comprising:

a CDKL5 polypeptide sequence, wherein the CDKL5 polypeptide sequence comprises SEQ ID NO:2 or SEQ ID NO: 16; and

a TATκ polypeptide sequence, wherein the TATκ polypeptide sequence comprises SEQ ID NO: 4, wherein the TATκ polypeptide is operatively coupled to the CDKL5 polypeptide.

2. The fusion protein of claim 1 , further comprising an Igk-chain leader sequence polypeptide, wherein the Igk-chain leader sequence is operatively coupled to the CDKL5 polypeptide.

3. The fusion protein of claim 1 , further comprising a reporter protein polypeptide, wherein the reporter protein polypeptide is operatively coupled to the CDKL5 polypeptide.

4. The fusion protein of claim 1 , further comprising a protein tag polypeptide, wherein the protein tag polypeptide is operatively coupled to the CDKL5 polypeptide.

5. The fusion protein of claim 1 , wherein the fusion protein has a polypeptide sequence comprising SEQ ID NO: 8, SEQ ID NO: 10, SEQ ID NO: 12, or SEQ ID NO: 14.

6. The fusion protein of claim 1 , wherein the fusion protein increases neurite growth, elongation, branch number, or branch density in the brain of a treated subject as compared to an untreated control subject.

7. The fusion protein of claim 1 , wherein the fusion protein reduces neuron apoptosis in the brain of a treated subject as compared to an untreated control subject.

8. A recombinant nucleic acid comprising:

a CDKL5 nucleic acid sequence, wherein the CDKL5 nucleic acid sequence comprises SEQ ID NO: 1 or SEQ ID NO: 15; and

a TATκ nucleic acid sequence, wherein the TATK nucleic acid sequence about 90% to about 100% sequence identity to SEQ ID NO: 3, wherein the TATK nucleic acid sequence is operatively coupled to the CDKL5 nucleic acid sequence.

9. The recombinant nucleic acid of claim 8 , wherein the nucleic acid encodes a fusion protein that increases neurite growth, elongation, branch number, or branch density in the brain of a treated subject as compared to an untreated control subject.

10. The recombinant nucleic acid of claim 8 , wherein the nucleic acid encodes a fusion protein that reduces neuron apoptosis in the brain of a treated subject as compared to an untreated control subject.

11. The recombinant nucleic acid of claim 8 , wherein the recombinant nucleic acid further comprises a nucleic acid sequence corresponding to a protein tag, wherein the nucleic acid sequence corresponding to a protein tag is operatively coupled to the CDKL5 nucleic acid sequence.

12. The recombinant nucleic acid of claim 11 , wherein the protein tag is selected from the group consisting of: chitin binding protein (CBP), maltose binding protein (MBP), glutathione-S-transferase (GST), poly(His), thioredoxin (TRX), poly(NANP), a FLAG-tag, V5-tag, Myc-tag, HA-tag, S-tag, SBP-Tag, Sftag 1, Softag 3, Tc tag, Xpress tag, Strep-tag, Isopeptag, Spy Tag, Ty tag, Biotin Carboxyl Carrier Protein (BCCP), Nus tag, and combinations thereof.

13. The recombinant nucleic acid of claim 8 , wherein the recombinant nucleic acid further comprises a nucleic acid sequence corresponding to a reporter protein, wherein the nucleic acid sequence corresponding to the reporter protein is operatively coupled to the CDKL5 nucleic acid sequence.

14. The recombinant nucleic acid of claim 13 , wherein the reporter protein is selected from the group consisting of: a fluorescent protein, beta-galactosidase, a luciferase protein, an antibiotic-resistance protein, p-glucuronidase, and alkaline phosphatase.

15. A vector comprising a recombinant nucleic acid of claim 8 .

16. The vector of claim 15 , wherein the nucleic acid encodes a fusion protein that increases neurite growth, elongation, branch number, or branch density in the brain of a treated subject as compared to an untreated control subject.

17. The vector of claim 15 , wherein the nucleic acid encodes a fusion protein that reduces neuron apoptosis in the brain of a treated subject as compared to an untreated control subject.

18. The vector of claim 15 , wherein the recombinant nucleic acid further comprises a nucleic acid sequence corresponding to a protein tag, wherein the nucleic acid sequence corresponding to a protein tag is operatively coupled to the CDKL5 nucleic acid sequence.

19. The vector of claim 18 , wherein the protein tag is selected from the group consisting of: chitin binding protein (CBP), maltose binding protein (MBP), glutathione-S-transferase (GST), poly(His), thioredoxin (TRX), poly(NANP), a FLAG-tag, V5-tag, Myc-tag, HA-tag, S-tag, SBP-Tag, Sftag 1, Softag 3, Tc tag, Xpress tag, Strep-tag, Isopeptag, Spy Tag, Ty tag, Biotin Carboxyl Carrier Protein (BCCP), Nus tag, and combinations thereof.

20. The vector of claim 15 , wherein the recombinant nucleic acid further comprises a nucleic acid sequence corresponding to a reporter protein, wherein the nucleic acid sequence corresponding to the reporter protein is operatively coupled to the CDKL5 nucleic acid sequence.

21. The vector of claim 15 , wherein the reporter protein is selected from the group consisting of: a fluorescent protein, beta-galactosidase, a luciferase protein, an antibiotic-resistance protein, p-glucuronidase, and alkaline phosphatase.

22. The vector of claim 15 , wherein the vector is a bacterial vector or an eukaryotic expression vector.

23. A composition comprising a recombinant nucleic acid of claim 8

and a pharmaceutically acceptable carrier.

24. The composition of claim 23 , wherein the nucleic acid encodes a fusion protein that increases neurite growth, elongation, branch number, or branch density in the brain of a treated subject as compared to an untreated control subject.

25. The composition of claim 23 , wherein the nucleic acid encodes a fusion protein that reduces neuron apoptosis in the brain of a treated subject as compared to an untreated control subject.

26. The composition of claim 23 , wherein the recombinant nucleic acid further comprises a nucleic acid sequence corresponding to a protein tag, wherein the nucleic acid sequence corresponding to a protein tag is operatively coupled to the CDKL5 nucleic acid sequence.

27. The composition of claim 26 , wherein the protein tag is selected from the group consisting of: chitin binding protein (CBP), maltose binding protein (MBP), glutathione-Stransferase (GST), poly(His), thioredoxin (TRX), poly(NANP), a FLAG-tag, V5-tag, Myc-tag, HA-tag, S-tag, SBP-Tag, Sftag 1, Softag 3, Tc tag, Xpress tag, Strep-tag, Isopeptag, Spy Tag, Ty tag, Biotin Carboxyl Carrier Protein (BCCP), Nus tag, and combinations thereof.

28. The composition of claim 23 , wherein the recombinant nucleic acid further comprises a nucleic acid sequence corresponding to a reporter protein, wherein the nucleic acid sequence corresponding to the reporter protein is operatively coupled to the CDKL5 nucleic acid sequence.

29. The composition of claim 28 , wherein the reporter protein is selected from the group consisting of: a fluorescent protein, beta-galactosidase, a luciferase protein, an antibiotic-resistance protein, p-glucuronidase, and alkaline phosphatase.

30. The composition of claim 23 , wherein the CDKL5 nucleic acid sequence and the TATK nucleic acid sequence are contained in a vector.

31. A method comprising:

transfecting cells with a recombinant nucleic acid, where the recombinant nucleic acid comprises:

a CDKL5 nucleic acid sequence, wherein the CDKL5 nucleic acid sequence comprises SEQ ID NO: 1 or SEQ ID NO: 15; and

a TATK nucleic acid sequence, wherein the TATK nucleic acid sequence about 90% to about 100% sequence identity to SEQ ID NO: 3, wherein the TATK nucleic acid sequence is operatively coupled to the CDKL5 nucleic acid sequence.

32. The method of claim 31 , wherein the recombinant nucleic acid is contained in a vector.

33. The method of claim 31 , wherein the cells comprise bacterial cells.

34. The method of claim 31 , wherein the cells comprise eukaryotic cells.

35. The method of claim 31 , further comprising the step of culturing the cells in media for a period of time.

36. The method of claim 35 , further comprising the step of collecting the media after the period of time.

37. The method of claim 36 , further comprising the step of harvesting a recombinant polypeptide from the collected media, where the recombinant polypeptide comprises a CDKL5 polypeptide operatively coupled to a TATκ polypeptide produced from the recombinant nucleic acid.

38. The method of claim 35 , further comprising the step of collecting the cells after the period of time.

39. The method of claim 38 , further comprising the steps of lysing the collected cells to form a cell lysate and harvesting a recombinant polypeptide from the cell lysate, where the recombinant polypeptide comprises a CDKL5 polypeptide operatively coupled to a TATK polypeptide produced from the recombinant nucleic acid.

Assignments (5)
RELEASE OF SECURITY INTEREST Recorded Apr 27, 2026
From: WILMINGTON TRUST, NATIONAL ASSOCIATION
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 075494/0030 →
SECURITY INTEREST Recorded Oct 6, 2023
From: AMICUS THERAPEUTICS, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 065177/0196 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 24, 2017
From: LACCONE, FRANCO
To: MEDICAL UNIVERSITY OF VIENNA
Reel/Frame 044280/0444 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 24, 2017
From: CIANI, ELISABETTA
To: ALMA MATER STUDIORUM - UNIVERSITÀ DI BOLOGNA
Reel/Frame 043934/0473 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 24, 2017
From: MEDICAL UNIVERSITY OF VIENNA
To: ALMA MATER STUDIORUM - UNIVERSITÀ DI BOLOGNA
Reel/Frame 043934/0766 →