US2023310657A1PendingUtilityA1

Modified mglur6 promoter and methods of use

71
Assignee: UNIV WAYNE STATEPriority: Mar 11, 2014Filed: Jan 11, 2023Published: Oct 5, 2023
Est. expiryMar 11, 2034(~7.6 yrs left)· nominal 20-yr term from priority
Inventors:Zhuo-Hua PanQi Long LuTushar H. GanjawalaJrgang Cheng
A61K 48/0058A61K 9/0048A61K 38/1787A61K 48/0075C07K 14/70571C12N 15/67C12N 15/85C12N 15/86C12N 2750/14143C12N 2830/008C12N 2830/42A61P 27/02
71
PatentIndex Score
0
Cited by
0
References
0
Claims

Abstract

The invention provides nucleic acids and nucleic acid expression vectors containing optimized mGluR6 promoters for expression of transgenes in the retina. The compositions and methods of the invention are useful for expression of gene products to preserve, improve, or restore phototransduction or vision.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . An adeno-associated virus (AAV) or a recombinant adeno-associated virus (rAAV) expression vector comprising a nucleic acid sequence encoding a capsid protein, a nucleic acid sequence of an mGluR6 promoter comprising SEQ ID NO: 6, or a sequence having at least 70% identity thereto, operably linked to a transgene encoding an opsin selected from the group consisting of a channelrhodopsin, a halorhodopsin, and a melanopsin. 
     
     
         2 . The expression vector of  claim 1 , wherein the expression vector comprises an mGluR6 promoter comprising a nucleic acid sequence having at least 80% identity to SEQ ID NO: 6. 
     
     
         3 . The expression vector of  claim 1 , wherein the expression vector comprises an mGluR6 promoter comprising a nucleic acid sequence having at least 90% identity to SEQ ID NO: 6. 
     
     
         4 . The expression vector of  claim 1 , wherein the expression vector comprises an mGluR6 promoter comprising the nucleic acid sequence of SEQ ID NO: 6. 
     
     
         5 . The expression vector of  claim 1 , wherein the vector further comprises an mGluR6 enhancer comprising the nucleic acid sequence of SEQ ID NO: 2, or a sequence having at least 70% identity thereto. 
     
     
         6 . The expression vector of  claim 1 , wherein the vector further comprises an mGluR6 enhancer comprising the nucleic acid sequence of SEQ ID NO: 2, or a sequence having at least 80% identity thereto. 
     
     
         7 . The expression vector of  claim 1 , wherein the vector further comprises an mGluR6 enhancer comprising the nucleic acid sequence of SEQ ID NO: 2, or a sequence having at least 90% identity thereto. 
     
     
         8 . The expression vector of  claim 1 , wherein the channelrhodopsin is selected from the group consisting of Chop1 (SEQ ID NO: 11), Chop2 (SEQ ID NO: 12), Chop2 315 aa fragment (SEQ ID NO: 13), vChR1 (SEQ ID NO: 14), and vChR2 (SEQ ID NO: 15), or an amino acid sequence having at least 90% identity thereto. 
     
     
         9 . The expression vector of  claim 1 , wherein the halorhodopsin is NpHR (SEQ ID NO: 16) or eNpHR. 
     
     
         10 . The expression vector of  claim 1 , wherein the melanopsin is a human melanopsin (SEQ ID NO: 18), or an amino acid sequence having at least 90% identity thereto. 
     
     
         11 . The expression vector of  claim 1 , wherein the vector is a recombinant AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, or AAV12 vector. 
     
     
         12 . The expression vector of  claim 11 , wherein the vector is a recombinant AAV2 vector. 
     
     
         13 . The expression vector of  claim 1 , wherein the amino acid sequence of the capsid protein is selected from the group consisting of SEQ ID Nos: 23-35. 
     
     
         14 . The expression vector of  claim 12 , wherein the capsid protein comprises a peptide selected from the group consisting of SEQ ID NO: 35, SEQ ID NO: 36, and SEQ ID NO: 37. 
     
     
         15 . An adeno-associated virus (AAV) or a recombinant adeno-associated virus (rAAV) expression vector comprising a nucleic acid sequence encoding a capsid protein, a nucleic acid sequence of an mGluR6 promoter comprising SEQ ID NO: 6, or a sequence having at least 70% identity thereto, operably linked to a transgene encoding a human melanopsin (SEQ ID NO: 18), or an amino acid sequence having at least 90% identity thereto. 
     
     
         16 . The expression vector of  claim 15 , wherein the vector further comprises an mGluR6 enhancer comprising the nucleic acid sequence of SEQ ID NO: 2, or a sequence having at least 70% identity thereto. 
     
     
         17 . A pharmaceutical composition comprising the expression vector of  claim 1 , and a pharmaceutically acceptable carrier or excipient. 
     
     
         18 . The pharmaceutical composition of  claim 17 , wherein the composition is formulated for intravitreal delivery. 
     
     
         19 . A pharmaceutical composition comprising the expression vector of  claim 15 , and a pharmaceutically acceptable carrier or excipient. 
     
     
         20 . The pharmaceutical composition of  claim 19 , wherein the composition is formulated for intravitreal delivery.

Join the waitlist — get patent alerts

Track US2023310657A1 — get alerts on status changes and closely related new filings.

We store only your email — no account needed. See our privacy policy.