US2002159979A1PendingUtilityA1

Adeno-associated virus materials and methods

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Assignee: CHILDRENS HOSPITAL INCPriority: Jun 6, 1994Filed: Feb 15, 2002Published: Oct 31, 2002
Est. expiryJun 6, 2014(expired)· nominal 20-yr term from priority
Inventors:Philip R. Johnson
A61K 39/00C12N 2750/14143C12N 2830/42C12N 7/00C12N 2710/10343C12N 2740/15034C12N 2740/15022A61K 48/00C07K 14/005C12N 2750/14122C12N 15/86A61K 2039/5256C12N 2750/14121
60
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Claims

Abstract

The present invention provides adeno-associated virus (AAV) materials and methods which are useful for DNA delivery to cells. More particularly, the invention provides recombinant AAV (rAAV) genomes, methods for packaging rAAV genomes, stable host cell lines producing rAAV and methods for delivering genes of interest to cells utilizing the rAAV. Particularly disclosed are rAAV useful in generating immunity to human immunodeficiency virus-1 and in therapeutic gene delivery for treatment of neurological disorders.

Claims

exact text as granted — not AI-modified
What is claimed is:  
     
         1 . A recombinant adeno-associated virus genome comprising adeno-associated virus inverted terminal repeats flanking DNA sequences encoding an immunodeficiency virus protein operably linked to promoter and polyadenylation sequences.  
     
     
         2 . The recombinant adeno-associated virus genome of  claim 1  comprising the cytomegalovirus (CMV) immediate early promoter, the rabbit β-globin intron, the human immunodeficiency virus rev/envelope sequences, and the rabbit β-globin polyadenylation signal.  
     
     
         3 . A recombinant adeno-associated virus genome comprising adeno-associated virus inverted terminal repeats flanking DNA sequences encoding a polypeptide selected from the group consisting of tyrosine hydroxylase, aromatic amino acid decarboxylase, nerve growth factor, brain derived neurotrophic factor, NT-3, NT-4/5, glial derived neurotrophic factor and fibroblast growth factor, wherein said DNA sequences are operably linked to promoter and polyadenylation sequences.  
     
     
         4 . A DNA vector comprising the recombinant adeno-associated virus genome of  claim 1  or  2 .  
     
     
         5 . The DNA vector according to  claim 4  which is vector pAAV/CMV/SIVrev-gp160/neo/rep-cap (ATCC 69637).  
     
     
         6 . A DNA vector comprising the recombinant adeno-associated virus genome of  claim 3 .  
     
     
         7 . A mammalian host cell stably transfected with a recombinant adeno-associated virus genome and with adeno-associated virus rep-cap genes.  
     
     
         8 . The mammalian host cell of  claim 7  wherein said recombinant adeno-associated virus genome is a recombinant adeno-associated virus genome according to  claim 1  or  2 .  
     
     
         9 . The mammalian host cell of  claim 8  which is HeLa cell line A64 (ATCC CRL 11639).  
     
     
         10 . A mammalian host cell stably transfected with a recombinant adeno-associated virus genome and with adeno-associated virus rep-cap genes, wherein said recombinant adeno-associated virus genome is a recombinant adeno-associated virus genome according to  claim 3 .  
     
     
         11 . A method for producing infectious recombinant adeno-associated virus comprising the step of infecting a host cell according to  claim 7  with a helper virus of adeno-associated virus.  
     
     
         12 . A method for producing infectious recombinant adeno-associated virus comprising the step of infecting a host cell according to  claim 8  with a helper virus of adeno-associated virus.  
     
     
         13 . A method for producing infectious recombinant adeno-associated virus comprising the step of infecting a host cell according to  claim 10  with a helper virus of adeno-associated virus.  
     
     
         14 . Infectious recombinant adeno-associated virus produced by the method of  claim 11 .  
     
     
         15 . Infectious recombinant adeno-associated virus produced by the method of  claim 12 .  
     
     
         16 . Infectious recombinant adeno-associated virus produced by the method of  claim 13 .  
     
     
         17 . A vaccine composition comprising the infectious recombinant adeno-associated virus of  claim 14 .  
     
     
         18 . A vaccine composition comprising the infectious recombinant adeno-associated virus of  claim 15 .  
     
     
         19 . A method for immunizing a host against human immunodeficiency virus comprising the step of administering an immunity-inducing dose of a vaccine composition according to  claim 17  to said host.  
     
     
         20 . A method for immunizing a host against human immunodeficiency virus comprising the step of administering an immunity-inducing dose of a vaccine composition according to  claim 18  to said host.  
     
     
         21 . A method for treating a neurodegenerative disorder comprising the step of administering a therapeutically effective dose of an infectious recombinant adeno-associated virus according to  claim 16  to a host exhibiting said neurodegenerative disoder.  
     
     
         22 . The method of  claim 21  wherein said neurodegenerative disorder is selected from the group consisting of Alzheimer's disease, Parkinson's disease, and Huntington's disease.  
     
     
         23 . The method of  claim 11  wherein said helper virus contains adeno-associated virus rep-cap genes inserted in its genome.  
     
     
         24 . The method of  claim 12  wherein said helper virus contains adeno-associated virus rep-cap genes inserted in its genome.  
     
     
         25 . The method of  claim 13  wherein said helper virus contains adeno-associated virus rep-cap genes inserted in its genome.

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