Title:
COMPOSITIONS AND METHODS FOR ENHANCED PARVOVIRUS TRANSDUCTION
Document Type and Number:
WIPO Patent Application WO/2011/097456
Kind Code:
A3
Abstract:
The present invention provides methods and compositions for enhanced transduction of an adeno-associated virus (AAV) vector comprising a heterologous nucleic acid of interest wherein the AAV vector genome is oversized relative to a wild type AAV genome by employing a proteasome inhibitor.
Inventors:
MONAHAN PAUL E (US)
SAMULSKI RICHARD JUDE (US)
SAMULSKI RICHARD JUDE (US)
Application Number:
PCT/US2011/023715
Publication Date:
December 08, 2011
Filing Date:
February 04, 2011
Export Citation:
Assignee:
UNIV NORTH CAROLINA (US)
MONAHAN PAUL E (US)
SAMULSKI RICHARD JUDE (US)
MONAHAN PAUL E (US)
SAMULSKI RICHARD JUDE (US)
International Classes:
C12N15/864; A61K31/517; A61K48/00; A61P7/04; A61P21/00; C12N15/33
Foreign References:
| US20090004145A1 | 2009-01-01 |
Other References:
ALLOCA ET AL.: "Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice", J CLIN INVEST., vol. 118, no. 5, May 2008 (2008-05-01), pages 1955 - 1964, XP002528465, DOI: doi:10.1172/JCI34316
"Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette", HUM GENE THER., vol. 19, no. 6, June 2008 (2008-06-01), pages 648 - 654
ATHANASOPOULOS ET AL.: "Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD).", GENE THER., vol. 11, no. SUPP.1, October 2004 (2004-10-01), pages S109 - S121, XP009048270, DOI: doi:10.1038/sj.gt.3302379
NEUKIRCHEN ET AL.: "The proteasome inhibitor bortezomib acts differently in combination with p53 gene transfer or cytotoxic chemotherapy on NSCLC cells.", CANCER GENE THER., vol. 14, no. 4, April 2007 (2007-04-01), pages 431 - 439
DONG ET AL.: "Characterization of genome integrity for oversized recombinant AAV vector.", MOL THER., vol. 18, no. 1, January 2010 (2010-01-01), pages 87 - 92, XP055183270, DOI: doi:10.1038/mt.2009.258
See also references of EP 2531604A4
"Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette", HUM GENE THER., vol. 19, no. 6, June 2008 (2008-06-01), pages 648 - 654
ATHANASOPOULOS ET AL.: "Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD).", GENE THER., vol. 11, no. SUPP.1, October 2004 (2004-10-01), pages S109 - S121, XP009048270, DOI: doi:10.1038/sj.gt.3302379
NEUKIRCHEN ET AL.: "The proteasome inhibitor bortezomib acts differently in combination with p53 gene transfer or cytotoxic chemotherapy on NSCLC cells.", CANCER GENE THER., vol. 14, no. 4, April 2007 (2007-04-01), pages 431 - 439
DONG ET AL.: "Characterization of genome integrity for oversized recombinant AAV vector.", MOL THER., vol. 18, no. 1, January 2010 (2010-01-01), pages 87 - 92, XP055183270, DOI: doi:10.1038/mt.2009.258
See also references of EP 2531604A4
Attorney, Agent or Firm:
MILLER, Mary, L. (P.A.P.O. Box 3742, Raleigh North Carolina, US)
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