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EP3177726
European procedure
Extract from the Register of European Patents
EP Citations: EP3177726
| Cited in | International search | Type: | Patent literature | Publication No.: | WO2013130824
[A] (SANGAMO BIOSCIENCES INC et al.) | Type: | Patent literature | Publication No.: | WO2014093701
[A] (BROAD INST INC et al.) | Type: | Non-patent literature | Publication information: | [A] M. GARRIGA-CANUT ET AL: "Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 109, no. 45, 10 October 2012 (2012-10-10), pages E3136 - E3145, XP055161220, ISSN: 0027-8424, DOI: 10.1073/pnas.1206506109 | DOI: | http://dx.doi.org/10.1073/pnas.1206506109 | Type: | Non-patent literature | Publication information: | [A] CONG LE ET AL: "In Vivo Genome Engineering With AAV Vector Carrying CRISPR-Cas9 System", MOLECULAR THERAPY, vol. 22, no. Suppl. 1, May 2014 (2014-05-01), & 17TH ANNUAL MEETING OF THE AMERICAN-SOCIETY-OF-GENE-AND-CELL-THERAPY (ASGCT); WASHINGTON, DC, USA; MAY 21 -24, 2014, pages S214, XP002734380 | Type: | Non-patent literature | Publication information: | [T] A. FISZER ET AL: "Oligonucleotide-based strategies to combat polyglutamine diseases", NUCLEIC ACIDS RESEARCH, vol. 42, no. 11, 21 May 2014 (2014-05-21), pages 6787 - 6810, XP055161218, ISSN: 0305-1048, DOI: 10.1093/nar/gku385 | DOI: | http://dx.doi.org/10.1093/nar/gku385 | Type: | Non-patent literature | Publication information: | [T] SAMUEL H. STERNBERG ET AL: "DNA interrogation by the CRISPR RNA-guided endonuclease Cas9", NATURE, vol. 507, no. 7490, 29 January 2014 (2014-01-29), pages 62 - 67, XP055161285, ISSN: 0028-0836, DOI: 10.1038/nature13011 | DOI: | http://dx.doi.org/10.1038/nature13011 | Type: | Non-patent literature | Publication information: | [T] MICHELE SIMONATO ET AL: "Progress in gene therapy for neurological disorders", NATURE REVIEWS NEUROLOGY, vol. 9, no. 5, 23 April 2013 (2013-04-23), pages 277 - 291, XP055161287, ISSN: 1759-4758, DOI: 10.1038/nrneurol.2013.56 | DOI: | http://dx.doi.org/10.1038/nrneurol.2013.56 | Type: | Non-patent literature | Publication information: | [T] C. ZUCCATO ET AL: "Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease", PHYSIOLOGICAL REVIEWS, vol. 90, no. 3, 1 July 2010 (2010-07-01), pages 905 - 981, XP055161189, ISSN: 0031-9333, DOI: 10.1152/physrev.00041.2009 | DOI: | http://dx.doi.org/10.1152/physrev.00041.2009 | Cited in | Examination | Type: | Non-patent literature | Publication information: | NICOLAS MERIENNE ET AL: "The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes", CELL REPORTS, vol. 20, no. 12, 1 September 2017 (2017-09-01), US, pages 2980 - 2991, XP055548036, ISSN: 2211-1247, DOI: 10.1016/j.celrep.2017.08.075 [DP] | DOI: | http://dx.doi.org/10.1016/j.celrep.2017.08.075 | Type: | Non-patent literature | Publication information: | ZOGHBI H Y ET AL: "Glutamine repeats and neurodegeneration", ANNUAL REVIEW OF NEUROSCIENCE, ANNUAL REVIEWS INC., PALO ALTO, CA, US, vol. 23, 1 January 2000 (2000-01-01), pages 217 - 247, XP002443799, ISSN: 0147-006X, DOI: 10.1146/ANNUREV.NEURO.23.1.217 | DOI: | http://dx.doi.org/10.1146/annurev.neuro.23.1.217 | Cited in | by applicant | Type: | Patent literature | Publication No.: | WO2013130824
| Type: | Patent literature | Publication No.: | WO2014093701
| Type: | Non-patent literature | Publication information: | SMITH; WATERMAN, ADVANCES IN APPLIED MATHEMATICS, vol. 2, 1981, pages 482 - 489 | Type: | Non-patent literature | Publication information: | W.R. PEARSON, METHODS IN ENZYMOLOGY, vol. 183, 1990, pages 63 - 98 | Type: | Non-patent literature | Publication information: | VONSATTEL JP; DIFIGLIA M: "Huntington disease", J NEUROPATH EXP NEUROL, vol. 57, 1998, pages 369 - 384 | Type: | Non-patent literature | Publication information: | ZUCCATO C; VALENZA M; CATTANEO E: "Molecular mechanisms and potential therapeutical targets in Huntington's disease", PHYSIOL REV, vol. 90, no. 3, 2010, pages 905 - 981 | Type: | Non-patent literature | Publication information: | ROSS CA; TABRIZI SJ: "Huntington's disease: from molecular pathogenesis to clinical treatment", LANCET NEURL, vol. 10, no. 1, 2011, pages 83 - 98 | Type: | Non-patent literature | Publication information: | FISZER A; KRZYZOSIAK WJ: "Oligonucleotide-based strategies to combat polyglutamine diseases", NUCLEIC ACIDS RES, vol. 42, no. 11, 2014, pages 6787 - 6810 | Type: | Non-patent literature | Publication information: | DROUET V ET AL.: "Sustained effects of nonallele-specific Huntingtin silencing", ANN NEUROL, vol. 65, no. 3, 2009, pages 276 - 285 | Type: | Non-patent literature | Publication information: | DROUET V ET AL.: "Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells", PLOS ONE, vol. 9, no. 6, 2014, pages E99341 | Type: | Non-patent literature | Publication information: | GARRIGA-CANUT M ET AL.: "Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice", PROC NATL ACAD SCI USA, vol. 109, no. 45, 2012, pages E3136 - 3145 | Type: | Non-patent literature | Publication information: | CONG L ET AL.: "Multiplex genome engineering using CRISPR/Cas systems", SCIENCE, vol. 339, no. 6121, 2013, pages 819 - 823 | Type: | Non-patent literature | Publication information: | JINEK M ET AL.: "RNA-programmed genome editing in human cells", ELIFE, vol. 2, 2013, pages E00471 | Type: | Non-patent literature | Publication information: | STERNBERG SH; REDDING S; JINEK M; GREENE EC; DOUDNA JA: "DNA interrogation by the CRISPR RNA-guided endonuclease Cas9", NATURE, vol. 507, no. 7490, 2014, pages 62 - 67 | Type: | Non-patent literature | Publication information: | MERIENNE N; LE DOUCE J; FAIVRE E; DEGLON N; BONVENTO G: "Efficient gene delivery and selective transduction of astrocytes in the mammalian brain using viral vectors", FRONT CELL NEUROSCI, vol. 7, 2013, pages 106 | Type: | Non-patent literature | Publication information: | SIMONATO M ET AL.: "Progress in gene therapy for neurological disorders", NAT REV NEUROL, vol. 9, no. 5, 2013, pages 277 - 291 | Type: | Non-patent literature | Publication information: | CARTIER N ET AL.: "Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy", SCIENCE, vol. 326, no. 5954, 2009, pages 818 - 823 | Type: | Non-patent literature | Publication information: | PALFI S ET AL.: "Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial", LANCET, vol. 383, no. 9923, 2014, pages 1138 - 1146 | Type: | Non-patent literature | Publication information: | WEINBERG MS; SAMULSKI RJ; MCCOWN TJ: "Adeno-associated virus (AAV) gene therapy for neurological disease", NEUROPHARMACOLOGY, vol. 69, 2013, pages 82 - 88 | Type: | Non-patent literature | Publication information: | NALDINI L ET AL.: "In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector", SCIENCE, vol. 272, no. 5259, 1996, pages 263 - 267 | Type: | Non-patent literature | Publication information: | MALI P ET AL.: "RNA-guided human genome engineering via Cas9", SCIENCE, vol. 339, no. 6121, 2013, pages 823 - 826 | Type: | Non-patent literature | Publication information: | JINEK M ET AL.: "A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity", SCIENCE, vol. 337, no. 6096, 2012, pages 816 - 821 | Type: | Non-patent literature | Publication information: | HIRANO M ET AL.: "Highly efficient retrograde gene transfer into motor neurons by a lentiviral vector pseudotyped with fusion glycoprotein", PLOS ONE, vol. 8, no. 9, 2013, pages E75896 | Type: | Non-patent literature | Publication information: | CASTLE MJ; GERSHENSON ZT; GILES AR; HOLZBAUR EL; WOLFE JH: "Adeno-Associated Virus Serotypes 1, 8, and 9 Share Conserved Mechanisms for Anterograde and Retrograde Axonal Transport", HUM GENE THER, vol. 25, no. 8, 2014, pages 705 - 720 | Type: | Non-patent literature | Publication information: | GRIMM D ET AL.: "In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses", J VIROL, vol. 82, no. 12, 2008, pages 5887 - 5911 | Type: | Non-patent literature | Publication information: | MELO SP ET AL.: "Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant", MOL THER, vol. 22, no. 4, 2014, pages 725 - 733 | Type: | Non-patent literature | Publication information: | DEGLON N ET AL.: "Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's disease", HUM GENE THER, vol. 11, no. 1, 2000, pages 179 - 190 | Type: | Non-patent literature | Publication information: | HOTTINGER AF; AZZOUZ M; DEGLON N; AEBISCHER P; ZURN AD: "Complete and long-term rescue oflesioned adult motoneurons by lentiviral-mediated expression of glial cell line-derived neurotrophic factor in the facial nucleus", J NEUROSC, vol. 20, no. 15, 2000, pages 5587 - 5593 | Type: | Non-patent literature | Publication information: | DULL T ET AL.: "A third-generation lentivirus vector with a conditional packaging system", J VIROL, vol. 72, no. 11, 1998, pages 8463 - 8471 | Type: | Non-patent literature | Publication information: | LOW K; AEBISCHER P; SCHNEIDER BL: "Direct and retrograde transduction of nigral neurons with AAV6, 8, and 9 and intraneuronal persistence of viral particles", HUM GENE THER, vol. 24, no. 6, 2013, pages 613 - 629 | Type: | Non-patent literature | Publication information: | DRINKUT A; TERESHCHENKO Y; SCHULZ JB; BAHR M; KUGLER S: "Efficient gene therapy for Parkinson's disease using astrocytes as hosts for localized neurotrophic factor delivery", MOL THER, vol. 20, no. 3, 2012, pages 534 - 543 | Type: | Non-patent literature | Publication information: | AURNHAMMER C ET AL.: "Universal real-time PCR for the detection and quantification of adeno-associated virus serotype 2-derived inverted terminal repeat sequences", HUM GENE THER METHODS, vol. 23, no. 1, 2012, pages 18 - 28 | Type: | Non-patent literature | Publication information: | DUJARDIN S ET AL.: "Ectosomes: a new mechanism for non-exosomal secretion of tau protein", PLOS ONE, vol. 9, no. 6, 2014, pages EL00760 | Type: | Non-patent literature | Publication information: | QIU P ET AL.: "Mutation detection using Surveyor nuclease", BIOTECHNIQUES, vol. 36, no. 4, 2004, pages 702 - 707 | Type: | Non-patent literature | Publication information: | RAN FA ET AL.: "Genome engineering using the CRISPR-Cas9 system", NAT PROTOC, vol. 8, no. 11, 2013, pages 2281 - 2308 |