IP Library › Granted Patent US 10,287,607
Granted Patent B2
US 10,287,607 · App. 16/153,401 · Granted May 14, 2019

Tissue selective transgene expression

Inventors: Stephanie Tagliatela (South San Francisco, CA); Andrew Young (South San Francisco, CA); Szu-Ying Chen (South San Francisco, CA); Kartik Ramamoorthi (South San Francisco, CA); David Oberkofler (South San Francisco, CA)
Assignee: Encoded Therapeutics, Inc.
C12N15/86A61K48/0058A61P25/28C12N15/8645A01K67/027A61K48/00A61P25/08C12N2750/14143C12N2750/14343C12N2840/007
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Quick Facts
Patent No.
US 10,287,607
App. No.
16/153,401
Granted
May 14, 2019
Kind
B2
Abstract

Provided herein are compositions and methods for selective expression of a transgene. Compositions and methods for selective expression of a transgene comprise one or more human regulatory elements, which, when operably linked to a transgene, can facilitate selective expression of a transgene (e.g., cell-type selective expression) in a target cell as compared to at least one or more non-target cells.

Claims (30)

1. A nucleic acid cassette comprising a regulatory element comprising SEQ ID NO: 1 operably linked to a heterologous transgene.

2. The nucleic acid cassette of claim 1 , wherein the heterologous transgene comprises a nucleic acid sequence encoding a DNA binding protein.

3. The nucleic acid cassette of claim 2 , wherein the DNA binding protein is a transcriptional activator that modulates an endogenous SCN1A gene.

4. The nucleic acid cassette of claim 1 , wherein the nucleic acid cassette is an adeno-associated virus (AAV) vector.

5. The nucleic acid cassette of claim 4 , wherein the AAV vector is AAV9 or scAAV9.

6. The nucleic acid cassette of claim 3 , wherein the nucleic acid cassette is an adeno-associated virus (AAV) vector.

7. The nucleic acid cassette of claim 6 , wherein the AAV vector is AAV9 or scAAV9.

8. The nucleic acid cassette of claim 1 , wherein the regulatory element comprises SEQ ID NO: 31.

9. The nucleic acid cassette of claim 8 , wherein the heterologous transgene comprises a nucleic acid sequence encoding a DNA binding protein.

10. The nucleic acid cassette of claim 9 , wherein the DNA binding protein is a transcriptional activator that modulates an endogenous SCN1A gene.

11. The nucleic acid cassette of claim 8 , wherein the nucleic acid cassette is an adeno-associated virus (AAV) vector.

12. The nucleic acid cassette of claim 11 , wherein the AAV vector is AAV9 or scAAV9.

13. The nucleic acid cassette of claim 10 , wherein the nucleic acid cassette is an adeno-associated virus (AAV) vector.

14. The nucleic acid cassette of claim 13 , wherein the AAV vector is AAV9.

15. The nucleic acid cassette of claim 13 , wherein the AAV vector is scAAV9.

16. A nucleic acid cassette comprising a regulatory element comprising a sequence with at least 95% sequence identity to SEQ ID NO: 1 operably linked to a heterologous transgene that results in selective expression in parvalbumin (PV) neurons in the CNS over one or more non-PV cells in the CNS.

17. The nucleic acid cassette of claim 16 , wherein the heterologous transgene comprises a nucleic acid sequence encoding a DNA binding protein.

18. The nucleic acid cassette of claim 17 , wherein the DNA binding protein is a transcriptional modulator of an endogenous gene.

19. The nucleic acid cassette of claim 17 , wherein the DNA binding protein comprises a zinc finger.

20. The nucleic acid cassette of claim 17 , wherein the DNA binding protein is linked to a transcriptional activator domain.

21. The nucleic acid cassette of claim 17 , wherein the DNA binding protein is a transcriptional activator that modulates an endogenous SCN1A gene.

22. The nucleic acid cassette of claim 16 , wherein the nucleic acid cassette is an adeno-associated virus (AAV) vector.

23. The nucleic acid cassette of claim 22 , wherein the AAV vector is AAV9 or scAAV9.

24. The nucleic acid cassette of claim 16 , wherein the regulatory element comprises a sequence with at least 95% sequence identity to SEQ ID NO: 31.

25. The nucleic acid cassette of claim 24 , wherein the heterologous transgene comprises a nucleic acid sequence encoding a DNA binding protein.

26. The nucleic acid cassette of claim 25 , wherein the DNA binding protein is a transcriptional activator that modulates an endogenous SCN1A gene.

27. The nucleic acid cassette of claim 24 , wherein the nucleic acid cassette is an adeno-associated virus (AAV) vector.

28. The nucleic acid cassette of claim 27 , wherein the AAV vector is AAV9 or scAAV9.

29. The nucleic acid cassette of claim 26 , wherein the nucleic acid cassette is an adeno-associated virus (AAV) vector.

30. The nucleic acid cassette of claim 29 , wherein the AAV vector is AAV9 or scAAV9.

Assignments (2)
CHANGE OF NAME Recorded Feb 21, 2019
From: ENCODED GENOMICS, INC.
To: ENCODED THERAPEUTICS, INC.
Reel/Frame 048410/0988 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 21, 2019
From: TAGLIATELA, STEPHANIE; YOUNG, ANDREW; CHEN, SZU-YING; RAMAMOORTHI, KARTIK; OBERKOFLER, DAVID
To: ENCODED GENOMICS, INC.
Reel/Frame 048402/0792 →
Continuity (3)
Continuation PCTUS2018025940 · Apr 3, 2018
Provisional Application 62480998 · Apr 3, 2017
Related Publication 20190024118A1 · Jan 24, 2019
Cited By (3)
US 12,467,064 US 12,516,350 US 12,692,516