US2007025970A1PendingUtilityA1

Vector system

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Assignee: OXFORD BIOMEDICA LTDPriority: Oct 6, 2000Filed: Jul 14, 2006Published: Feb 1, 2007
Est. expiryOct 6, 2020(expired)· nominal 20-yr term from priority
Inventors:Alan KingsmanNicholas MazarakisEnca Martin-RendonMimoun AzzouzJonathan Rohll
C12N 2740/15043A61P 25/28C12N 2840/206C12N 2830/48A61P 25/16C12N 9/0071C12N 15/86A61K 48/00
54
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Claims

Abstract

The present invention relates to retroviral vector genomes and to vector systems comprising such genomes. In particular the present invention relates to a retroviral vector genome comprising two or more NOIs operably linked by one or more Internal Ribosome Entry Site(s); a lentiviral vector genome comprising two or more NOIs suitable for treating a neurodegenerative disorder; and a lentiviral vector genome which encodes tyrosine hydroxylase, GTP-cyclohydrolase I and optionally Aromatic Amino Acid Dopa Decarboxylase.

Claims

exact text as granted — not AI-modified
1 - 71 . (canceled)  
     
     
         72 . A method for treating Parkinson's disease in a subject in need thereof, comprising administering into the brain of the subject a lentiviral vector comprising three nucleotides of interest (NOIs) operably linked by one or more Internal Ribosome Entry Site(s) (IRES), wherein the NOIs encode tyrosine hydroxylase (TH), GTP-cyclohydrolase I (GTP-CH1) and aromatic amino acid dopa decarboxylase (AADC), and wherein the three NOIs are expressed to stimulate dopamine synthesis in the brain, thereby treating the subject.  
     
     
         73 . The method according to  claim 72 , wherein the lentiviral vector is an HIV lentiviral vector.  
     
     
         74 . The method according to  claim 72 , wherein the lentiviral vector is a non-primate lentiviral vector.  
     
     
         75 . The method according to  claim 74 , wherein the lentiviral vector is an EIAV lentiviral vector.  
     
     
         76 . The method according to  claim 72 , wherein at least one of the NOIs is operably linked to a promoter or promoter elements.  
     
     
         77 . The method according to  claim 72 , wherein the lentiviral vector further comprises a post-transcriptional regulatory element or a translational enhancer.  
     
     
         78 . The method according to  claim 72 , wherein at least one of the NOIs is codon optimized.  
     
     
         79 . The method according to  claim 72 , wherein the lentiviral vector is pseudotyped with at least part of a heterologous env protein.  
     
     
         80 . The method according to  claim 79 , wherein the heterologous env protein is Rabies-G or VSV-G.  
     
     
         81 . The method according to  claim 72 , wherein the IRES is a viral IRES.  
     
     
         82 . The method according to  claim 81 , wherein the viral IRES is from a picornavirus.  
     
     
         83 . The method according to  claim 82 , wherein the picornavirus is encephalomyocarditis virus (EMCV) or poliovirus (PV).  
     
     
         84 . The method according to  claim 72 , wherein the lentiviral vector comprises a self-inactivating lentiviral vector genome.  
     
     
         85 . A pharmaceutical composition comprising a lentiviral vector comprising three nucleotides of interest (NOIs) operably linked by one or more Internal Ribosome Entry Site(s) (IRES), wherein the NOIs encode tyrosine hydroxylase (TH), GTP-cyclohydrolase I (GTP-CH1) and aromatic amino acid dopa decarboxylase (AADC).  
     
     
         86 . The pharmaceutical composition of  claim 85 , wherein the lentiviral vector is an HIV lentiviral vector.  
     
     
         87 . The pharmaceutical composition of  claim 85 , wherein the lentiviral vector is a non-primate lentiviral vector.  
     
     
         88 . The pharmaceutical composition of  claim 87 , wherein the lentiviral vector is an EIAV lentiviral vector.  
     
     
         89 . The pharmaceutical composition of  claim 85 , wherein at least one of the NOIs is operably linked to a promoter or promoter elements.  
     
     
         90 . The pharmaceutical composition of  claim 85 , wherein the lentiviral vector further comprises a post-transcriptional regulatory element or a translational enhancer.  
     
     
         91 . The pharmaceutical composition of  claim 85 , wherein at least one of the NOIs is codon optimized.  
     
     
         92 . The pharmaceutical composition of  claim 85 , wherein the lentiviral vector is pseudotyped with at least part of a heterologous env protein.  
     
     
         93 . The pharmaceutical composition of  claim 92 , wherein the heterologous env protein is Rabies-G or VSV-G.  
     
     
         94 . The pharmaceutical composition of  claim 85 , wherein the IRES is a viral IRES.  
     
     
         95 . The pharmaceutical composition of  claim 94 , wherein the viral IRES is from a picornavirus.  
     
     
         96 . The pharmaceutical composition of  claim 95 , wherein the picornavirus is encephalomyocarditis virus (EMCV) or poliovirus (PV).  
     
     
         97 . The pharmaceutical composition of  claim 85 , wherein the lentiviral vector comprises a self-inactivating lentiviral vector genome.

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