(19)
(11) EP 3 280 451 B8

(12) CORRECTED EUROPEAN PATENT SPECIFICATION
Note: Bibliography reflects the latest situation

(15) Correction information:
Corrected version no 1 (W1 B1)

(48) Corrigendum issued on:
25.09.2019 Bulletin 2019/39

(45) Mention of the grant of the patent:
14.08.2019 Bulletin 2019/33

(21) Application number: 16717228.7

(22) Date of filing: 07.04.2016
(51) International Patent Classification (IPC): 
A61K 48/00(2006.01)
C12N 15/864(2006.01)
A61P 25/28(2006.01)
(86) International application number:
PCT/US2016/026524
(87) International publication number:
WO 2016/164642 (13.10.2016 Gazette 2016/41)

(54)

VIRAL GENE THERAPY AS TREATMENT FOR CHOLESTEROL STORAGE DISEASE OR DISORDER

VIRALE GENTHERAPIE ZUR BEHANDLUNG VON CHOLESTERINSPEICHERUNGSERKRANKUNG ODER -STÖRUNG

THÉRAPIE GÉNIQUE VIRALE À UTILISER EN TANT QUE TRAITEMENT POUR UNE MALADIE OU UN TROUBLE ASSOCIÉ AU STOCKAGE DU CHOLESTÉROL


(84) Designated Contracting States:
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

(30) Priority: 08.04.2015 US 201562144702 P

(43) Date of publication of application:
14.02.2018 Bulletin 2018/07

(73) Proprietor: The United States of America, as represented by the Secretary, Department of Health and Human Services
Maryland 20892-7660 (US)

(72) Inventors:
  • VENDITTI, Charles, P.
    Kensington, MD 20895 (US)
  • PAVAN, William, J.
    Derwood, MD 20855 (US)
  • CHANDLER, Randy
    Washington, DC 20009 (US)

(74) Representative: Huenges, Martin 
Maiwald Patentanwalts- und Rechtsanwaltsgesellschaft mbH Elisenhof Elisenstraße 3
80335 München
80335 München (DE)


(56) References cited: : 
WO-A1-2009/043936
WO-A2-2012/061744
WO-A1-2011/020118
   
  • C.A. PAUL ET AL: "Adenovirus expressing an NPC1-GFP fusion gene corrects neuronal and nonneuronal defects associated with Niemann pick type C disease", JOURNAL OF NEUROSCIENCE RESEARCH., vol. 81, no. 5, 1 September 2005 (2005-09-01), pages 706-719, XP055295101, US ISSN: 0360-4012, DOI: 10.1002/jnr.20592
  • Donald B Arnold ET AL: "A calcium responsive element that regulates expression of two calcium binding proteins in Purkinje cells (transcription͞biolistic transfection͞Ca 2? responses͞calbindin͞calmodulin II) Communicated by", Neurobiology May, 1 August 1997 (1997-08-01), pages 8842-8847, XP055295089, Retrieved from the Internet: URL:http://www.pnas.org/content/94/16/8842 .full.pdf [retrieved on 2016-08-11]
  • T D BARBARA NGUYEN-VU ET AL: "Cerebellar Purkinje cell activity drives motor learning", NATURE NEUROSCIENCE, vol. 16, no. 12, 27 October 2013 (2013-10-27), pages 1734-1736, XP55295269, US ISSN: 1097-6256, DOI: 10.1038/nn.3576
   
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