EP 3389671 A4 20190717 - ANTISENSE OLIGOMERS FOR TREATMENT OF ALAGILLE SYNDROME
Title (en)
ANTISENSE OLIGOMERS FOR TREATMENT OF ALAGILLE SYNDROME
Title (de)
ANTISENSE-OLIGOMERE ZUR BEHANDLUNG DES ALAGILLE-SYNDROMS
Title (fr)
OLIGOMÈRES ANTISENS POUR LE TRAITEMENT DU SYNDROME D'ALAGILLE
Publication
Application
Priority
- US 201562267210 P 20151214
- US 2016066414 W 20161213
Abstract (en)
[origin: WO2017106210A1] Provided herein are methods and compositions for increasing the expression of JAGl, and for treating a subject in need thereof, e.g., a subject with deficient JAGl protein expression or a subject having Alagille syndrome (ALGS).
IPC 8 full level
C12N 15/113 (2010.01); A61K 31/7088 (2006.01); A61P 1/16 (2006.01); C12N 15/11 (2006.01); C12Q 1/68 (2018.01)
CPC (source: EP)
A61K 31/7088 (2013.01); A61P 1/16 (2018.01); A61P 21/04 (2018.01); A61P 43/00 (2018.01); C12N 15/111 (2013.01); C12N 15/113 (2013.01); C12Q 1/68 (2013.01); C12Q 1/6883 (2013.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2320/33 (2013.01); C12Q 2600/156 (2013.01)
C-Set (source: EP)
Citation (search report)
- [XI] WO 2007048628 A2 20070503 - MAX PLANCK GESELLSCHAFT [DE], et al
- [X] WO 2007048629 A2 20070503 - MAX PLANCK GESELLSCHAFT [DE], et al
- [X] WO 2005049651 A2 20050602 - UNIV BELFAST [GB], et al
- [X] M. YAMAMOTO ET AL: "Mib-Jag1-Notch signalling regulates patterning and structural roles of the notochord by controlling cell-fate decisions", DEVELOPMENT, vol. 137, no. 15, 23 June 2010 (2010-06-23), GB, pages 2527 - 2537, XP055587656, ISSN: 0950-1991, DOI: 10.1242/dev.051011
- [A] PETER D TURNPENNY ET AL: "Alagille syndrome: pathogenesis, diagnosis and management", EUROPEAN JOURNAL OF HUMAN GENETICS., vol. 20, no. 3, 21 September 2011 (2011-09-21), CH, pages 251 - 257, XP055590188, ISSN: 1018-4813, DOI: 10.1038/ejhg.2011.181
- [A] NANCY B. SPINNER ET AL: "Jagged1 mutations in Alagille syndrome", HUMAN MUTATION, vol. 17, no. 1, 22 December 2000 (2000-12-22), US, pages 18 - 33, XP055587899, ISSN: 1059-7794, DOI: 10.1002/1098-1004(2001)17:1<18::AID-HUMU3>3.0.CO;2-T
- [A] GIUSEPPE PILIA ET AL: "Jagged-1 mutation analysis in Italian Alagille syndrome patients", HUMAN MUTATION, vol. 14, no. 5, 22 October 1999 (1999-10-22), US, pages 394 - 400, XP055587922, ISSN: 1059-7794, DOI: 10.1002/(SICI)1098-1004(199911)14:5<394::AID-HUMU5>3.0.CO;2-1
- [A] LITING LI ET AL: "JAG1 Mutation Spectrum and Origin in Chinese Children with Clinical Features of Alagille Syndrome", PLOS ONE, vol. 10, no. 6, 15 June 2015 (2015-06-15), pages e0130355, XP055587896, DOI: 10.1371/journal.pone.0130355
- [A] JANA KRALOVICOVA ET AL: "Optimal antisense target reducing INS intron 1 retention is adjacent to a parallel G quadruplex", NUCLEIC ACIDS RESEARCH, vol. 42, no. 12, 17 June 2014 (2014-06-17), pages 8161 - 8173, XP055211568, ISSN: 0305-1048, DOI: 10.1093/nar/gku507
- [A] RYSZARD KOLE ET AL: "RNA therapeutics: beyond RNA interference and antisense oligonucleotides", NATURE REVIEWS DRUG DISCOVERY, vol. 11, no. 2, 20 January 2012 (2012-01-20), pages 125 - 40, XP055138361, ISSN: 1474-1776, DOI: 10.1038/nrd3625
Citation (examination)
- US 2004102401 A1 20040527 - DEAN NICHOLAS M [US], et al
- See also references of WO 2017106210A1
Designated contracting state (EPC)
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
DOCDB simple family (publication)
WO 2017106210 A1 20170622; CA 3005245 A1 20170622; EP 3389671 A1 20181024; EP 3389671 A4 20190717; JP 2018538288 A 20181227; JP 2022106803 A 20220720
DOCDB simple family (application)
US 2016066414 W 20161213; CA 3005245 A 20161213; EP 16876499 A 20161213; JP 2018529221 A 20161213; JP 2022069164 A 20220420