WO2007127428A3 - Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery - Google Patents

Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery Download PDF

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Publication number
WO2007127428A3
WO2007127428A3 PCT/US2007/010338 US2007010338W WO2007127428A3 WO 2007127428 A3 WO2007127428 A3 WO 2007127428A3 US 2007010338 W US2007010338 W US 2007010338W WO 2007127428 A3 WO2007127428 A3 WO 2007127428A3
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WO
WIPO (PCT)
Prior art keywords
type
self
host cells
promoter
vector
Prior art date
Application number
PCT/US2007/010338
Other languages
French (fr)
Other versions
WO2007127428A8 (en
WO2007127428A2 (en
Inventor
Barry John Byrne
Sanford Leon Boye
William W Hauswirth
Original Assignee
Univ Florida
Barry John Byrne
Sanford Leon Boye
William W Hauswirth
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Florida, Barry John Byrne, Sanford Leon Boye, William W Hauswirth filed Critical Univ Florida
Priority to US12/298,845 priority Critical patent/US8298818B2/en
Publication of WO2007127428A2 publication Critical patent/WO2007127428A2/en
Publication of WO2007127428A3 publication Critical patent/WO2007127428A3/en
Publication of WO2007127428A8 publication Critical patent/WO2007127428A8/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

Abstract

The present inventors concerns vectors carrying a truncated chimeric CMV- chicken β-actin (smCBA) promoter in which the hybrid chicken β-actin/rabbit β-globin intron is greatly shortened, and their use to deliver to an operatively linked polynucleotide to host cells in vitro or in vivo, resulting in expression of the polynucleotide in the host cells. In one embodiment, the vector carrying the smCBA promoter is administered to the eye. In another embodiment, the vector carrying the smCBA promoter is a self- complementary adeno-associated virus (AAV). The AAV vector may be of any serotype (e.g., type 1, type 2, type 3, type 4, type 5, type 6, type 7, type 8, type 9, type 10). In another embodiment, a self-complementary vector carrying the smCBA promoter is administered to the eye. Another aspect of the invention concerns host cells carrying a vector of the invention. Another aspect of the invention concerns pharmaceutical composition comprising the vectors or host cells of the invention, and a pharmaceutically acceptable carrier.
PCT/US2007/010338 2006-04-28 2007-04-27 Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery WO2007127428A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
US12/298,845 US8298818B2 (en) 2006-04-28 2007-04-27 Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US79575906P 2006-04-28 2006-04-28
US60/795,759 2006-04-28

Publications (3)

Publication Number Publication Date
WO2007127428A2 WO2007127428A2 (en) 2007-11-08
WO2007127428A3 true WO2007127428A3 (en) 2008-02-28
WO2007127428A8 WO2007127428A8 (en) 2008-04-10

Family

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Family Applications (1)

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PCT/US2007/010338 WO2007127428A2 (en) 2006-04-28 2007-04-27 Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery

Country Status (2)

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US (1) US8298818B2 (en)
WO (1) WO2007127428A2 (en)

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Also Published As

Publication number Publication date
WO2007127428A8 (en) 2008-04-10
US8298818B2 (en) 2012-10-30
US20100069467A1 (en) 2010-03-18
WO2007127428A2 (en) 2007-11-08

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