US2023374545A1PendingUtilityA1

Aav capsid designs

Assignee: UNIV MASSACHUSETTSPriority: Oct 13, 2016Filed: Jan 5, 2023Published: Nov 23, 2023
Est. expiryOct 13, 2036(~10.2 yrs left)· nominal 20-yr term from priority
C12N 15/8509C07K 14/005A61K 48/00C12N 15/86C12N 15/861C07K 14/015A61K 35/761A01K 67/0275C12N 2750/14122C12N 2750/14123A01K 2267/03A01K 2217/05C12N 2750/14143C12N 2750/14145A61P 1/00A61P 1/16A61P 1/18A61P 11/00A61P 13/02A61P 13/12A61P 15/00A61P 19/06A61P 21/04A61P 25/00A61P 25/16A61P 27/02A61P 35/00A61P 3/06A61P 3/10A61P 37/02A61P 43/00A61P 7/02A61P 9/00A61P 9/04A61P 9/10C12N 2015/8518
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Claims

Abstract

The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

Claims

exact text as granted — not AI-modified
1 . A recombinant expression vector comprising a nucleic acid encoding a polypeptide having a sequence selected from the group consisting of: SEQ ID NOs: 21-62, 67-799, 802-1725, and 1729-1986. 
     
     
         2 . An isolated AAV capsid protein comprising an amino acid sequence selected from the group consisting of: SEQ ID NOs: 21-62, 67-799, 802-1725, and 1729-1986. 
     
     
         3 .- 7 . (canceled) 
     
     
         8 . An recombinant expression vector comprising a nucleic acid sequence encoding the isolated AAV capsid protein of  claim 2 . 
     
     
         9 .- 10 . (canceled) 
     
     
         11 . A recombinant AAV (rAAV) comprising the isolated AAV capsid protein of  claim 2 . 
     
     
         12 .- 13 . (canceled) 
     
     
         14 . A host cell containing a recombinant expression vector of  claim 8 . 
     
     
         15 .- 17 . (canceled) 
     
     
         17 . A method for delivering at least one transgene to a subject comprising administering a rAAV of  claim 11  to a subject, wherein the rAAV comprises the at least one transgene, and wherein the rAAV infects cells of a target tissue of the subject. 
     
     
         18 . A method for generating a somatic transgenic animal model comprising administering a recombinant rAAV of  claim 11  to a non-human animal, wherein the rAAV comprises at least one transgene, and wherein the rAAV infects cells of a target tissue of the non-human animal. 
     
     
         19 . The method of  claim 17 , wherein the at least one transgene is a protein coding gene or encodes a small interfering nucleic acid. 
     
     
         20 .- 23 . (canceled) 
     
     
         24 . The method of  claim 17 , wherein the target tissue is liver, central nervous system (CNS), bone, cartilage, ocular, gastrointestinal, respiratory, breast, pancreas, urinary tract, or uterine tissue. 
     
     
         25 .- 28 . (canceled) 
     
     
         29 . The method of  claim 17 , wherein the rAAV is administered intravenously, transdermally, intraocularly, intrathecally, orally, intramuscularly, subcutaneously, intranasally, or by inhalation. 
     
     
         30 . The method of  claim 17 , wherein the subject is selected from a human, a mouse, a rat, a rabbit, a dog, a cat, a sheep, a pig, and a non-human primate. 
     
     
         31 .- 35 . (canceled) 
     
     
         36 . A kit comprising:
 a container housing a recombinant AAV having an isolated AAV capsid protein of  claim 2 .   
     
     
         37 . (canceled) 
     
     
         38 . The isolated AAV capsid protein of  claim 2 , wherein the capsid protein is a VP1 capsid protein, a VP2 capsid protein, or a VP3 capsid protein. 
     
     
         39 .- 41 . (canceled) 
     
     
         42 . The recombinant expression vector of  claim 1 , wherein the nucleic acid encodes a V1 capsid protein, a VP2 capsid protein, or a VP3 capsid protein. 
     
     
         43 .- 50 . (canceled) 
     
     
         51 . The recombinant expression vector of  claim 1  comprising a nucleic acid encoding a polypeptide having a sequence selected from the group consisting of: SEQ ID NOs: 124, 149, 161, 175, and 358. 
     
     
         52 . The recombinant expression vector of  claim 1  comprising a nucleic acid encoding a polypeptide having a sequence selected from the group consisting of: SEQ ID NOs: 149, 152, 157, and 182. 
     
     
         53 . The recombinant expression vector of  claim 1  comprising a nucleic acid encoding a polypeptide having a sequence selected from the group consisting of: SEQ ID NOs: 46, 56, 67, 81, 224, 326, and 358. 
     
     
         54 . The isolated AAV capsid protein of  claim 2  comprising a nucleic acid encoding a polypeptide having a sequence selected from the group consisting of: SEQ ID NOs: 124, 149, 161, 175, and 358. 
     
     
         55 . The isolated AAV capsid protein of  claim 2  comprising a nucleic acid encoding a polypeptide having a sequence selected from the group consisting of: SEQ ID NOs: 149, 152, 157, and 182. 
     
     
         56 . The isolated AAV capsid protein of  claim 2  comprising a nucleic acid encoding a polypeptide having a sequence selected from the group consisting of: SEQ ID NOs: 46, 56, 67, 81, 224, 326, and 358.

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