Title:
ENHANCED DELIVERY OF VIRAL PARTICLES TO THE STRIATUM AND CORTEX
Document Type and Number:
WIPO Patent Application WO/2016/130591
Kind Code:
A3
Abstract:
Provided herein are novel methods for delivering recombinant adeno-associated viral (rAAV) particles to the central nervous system of a mammal (e.g., a human). In aspects, the methods involve administering rAAV particles containing a heterologous nucleic acid to the striatum and causing expression of the heterologous nucleic acid in at least the cerebral cortex and the striatum of the mammal.
Inventors:
STANEK LISA M (US)
SHIHABUDDIN LAMYA (US)
SHIHABUDDIN LAMYA (US)
Application Number:
PCT/US2016/017210
Publication Date:
November 10, 2016
Filing Date:
February 09, 2016
Export Citation:
Assignee:
GENZYME CORP (US)
International Classes:
C12N15/86; A61K48/00
Domestic Patent References:
| WO2012109667A1 | 2012-08-16 | |||
| WO2015168666A2 | 2015-11-05 | |||
| WO1999061066A2 | 1999-12-02 | |||
| WO2010088560A1 | 2010-08-05 |
Other References:
SAN SEBASTIAN W ET AL: "Adeno-associated virus type 6 is retrogradely transported in the non-human primate brain", GENE THERAPY, vol. 20, no. 12, 26 December 2013 (2013-12-26), GB, pages 1178 - 1183, XP055277697, ISSN: 0969-7128, DOI: 10.1038/gt.2013.48
VITE C H ET AL: "Adeno-Associated Virus Vector-Mediated Transduction in the Cat Brain", GENE THERAPY, NATURE PUBLISHING GROUP, GB, vol. 10, 1 January 2003 (2003-01-01), pages 1874 - 1881, XP003002610, ISSN: 0969-7128
STANEK L ET AL: "Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease", HUMAN GENE THERAPY, vol. 25, no. 5, 1 May 2014 (2014-05-01), US, pages 461 - 474, XP055277689, ISSN: 1043-0342, DOI: 10.1089/hum.2013.200
SONDHI D ET AL: "AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL", GENE THERAPY, vol. 12, no. 22, 1 November 2005 (2005-11-01), GB, pages 1618 - 1632, XP055304187, ISSN: 0969-7128, DOI: 10.1038/sj.gt.3302549
SANFTNER L M ET AL: "AAV2-mediated gene delivery to monkey putamen: Evaluation of an infusion device and delivery parameters", EXPERIMENTAL NEUROLOGY, ACADEMIC PRESS, NEW YORK, NY, US, vol. 194, no. 2, 1 August 2005 (2005-08-01), pages 476 - 483, XP004977564, ISSN: 0014-4886, DOI: 10.1016/J.EXPNEUROL.2005.03.007
ZHANG S H ET AL: "Transduction of striatum and cortex tissues by adeno-associated viral vectors produced by herpes simplex virus- and baculovirus-based methods", JOURNAL OF VIROLOGICAL METHODS, ELSEVIER BV, NL, vol. 179, no. 1, 4 October 2011 (2011-10-04), pages 276 - 280, XP028355367, ISSN: 0166-0934, [retrieved on 20111012], DOI: 10.1016/J.JVIROMET.2011.10.004
RAMASWAMY S ET AL: "Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease", NEUROBIOLOGY OF DISEASE, BLACKWELL SCIENTIFIC PUBLICATIONS, OXFORD, GB, vol. 34, no. 1, 1 April 2009 (2009-04-01), pages 40 - 50, XP026031297, ISSN: 0969-9961, [retrieved on 20090324], DOI: 10.1016/J.NBD.2008.12.005
HADACZEK P ET AL: "Convection-Enhanced Delivery of Adeno-Associated Virus Type 2 (AAV2) into the Striatum and Transport of AAV2 Within Monkey Brain", HUMAN GENE THERAPY, vol. 17, no. 3, 1 March 2006 (2006-03-01), US, pages 291 - 302, XP055304207, ISSN: 1043-0342, DOI: 10.1089/hum.2006.17.291
VITE C H ET AL: "Adeno-Associated Virus Vector-Mediated Transduction in the Cat Brain", GENE THERAPY, NATURE PUBLISHING GROUP, GB, vol. 10, 1 January 2003 (2003-01-01), pages 1874 - 1881, XP003002610, ISSN: 0969-7128
STANEK L ET AL: "Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease", HUMAN GENE THERAPY, vol. 25, no. 5, 1 May 2014 (2014-05-01), US, pages 461 - 474, XP055277689, ISSN: 1043-0342, DOI: 10.1089/hum.2013.200
SONDHI D ET AL: "AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL", GENE THERAPY, vol. 12, no. 22, 1 November 2005 (2005-11-01), GB, pages 1618 - 1632, XP055304187, ISSN: 0969-7128, DOI: 10.1038/sj.gt.3302549
SANFTNER L M ET AL: "AAV2-mediated gene delivery to monkey putamen: Evaluation of an infusion device and delivery parameters", EXPERIMENTAL NEUROLOGY, ACADEMIC PRESS, NEW YORK, NY, US, vol. 194, no. 2, 1 August 2005 (2005-08-01), pages 476 - 483, XP004977564, ISSN: 0014-4886, DOI: 10.1016/J.EXPNEUROL.2005.03.007
ZHANG S H ET AL: "Transduction of striatum and cortex tissues by adeno-associated viral vectors produced by herpes simplex virus- and baculovirus-based methods", JOURNAL OF VIROLOGICAL METHODS, ELSEVIER BV, NL, vol. 179, no. 1, 4 October 2011 (2011-10-04), pages 276 - 280, XP028355367, ISSN: 0166-0934, [retrieved on 20111012], DOI: 10.1016/J.JVIROMET.2011.10.004
RAMASWAMY S ET AL: "Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease", NEUROBIOLOGY OF DISEASE, BLACKWELL SCIENTIFIC PUBLICATIONS, OXFORD, GB, vol. 34, no. 1, 1 April 2009 (2009-04-01), pages 40 - 50, XP026031297, ISSN: 0969-9961, [retrieved on 20090324], DOI: 10.1016/J.NBD.2008.12.005
HADACZEK P ET AL: "Convection-Enhanced Delivery of Adeno-Associated Virus Type 2 (AAV2) into the Striatum and Transport of AAV2 Within Monkey Brain", HUMAN GENE THERAPY, vol. 17, no. 3, 1 March 2006 (2006-03-01), US, pages 291 - 302, XP055304207, ISSN: 1043-0342, DOI: 10.1089/hum.2006.17.291
Attorney, Agent or Firm:
DONAHUE, Brian, A. et al. (755 Page Mill RoadPalo Alto, CA, US)
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