(19)
(11) EP 3 298 134 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:
21.06.2023 Bulletin 2023/25

(45) Mention of the grant of the patent:
10.05.2023 Bulletin 2023/19

(21) Application number: 16718840.8

(22) Date of filing: 15.04.2016
(51) International Patent Classification (IPC): 
C12N 9/22(2006.01)
A61P 27/00(2006.01)
A61P 25/00(2006.01)
 C12N 15/00(2006.01)
A61P 21/00(2006.01)
A61P 3/00(2006.01)
(52) Cooperative Patent Classification (CPC):
A61K 31/7088; A61K 31/7105; C12N 2750/14143; A61K 38/465; C12N 9/22; A61P 11/00; A61P 13/02; A61P 13/12; A61P 21/00; A61P 21/02; A61P 21/04; A61P 25/00; A61P 25/02; A61P 25/28; A61P 27/00; A61P 27/02; A61P 27/12; A61P 3/00; A61P 3/12; A61P 35/00; A61P 35/02; A61P 3/08; A61P 37/02; A61P 43/00; A61P 5/00; A61P 7/02; A61P 7/04; A61P 7/06; A61P 9/00
 
C-Sets:
  1. A61K 31/7105, A61K 2300/00;
  2. A61K 31/7088, A61K 2300/00;
  3. A61K 38/465, A61K 2300/00;
(86) International application number:
PCT/US2016/027987
(87) International publication number:
WO 2016/186772 (24.11.2016 Gazette 2016/47)

(54)

GENE EDITING OF DEEP INTRONIC MUTATIONS

GEN-EDITING VON TIEFEN INTRONISCHEN MUTATIONEN

ÉDITION GÉNIQUE DE MUTATIONS INTRONIQUES PROFONDES

(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
Designated Extension States:
BA ME

(30) Priority: 16.05.2015 US 201562162720 P

(43) Date of publication of application:
28.03.2018 Bulletin 2018/13

(73) Proprietor: Genzyme Corporation
Cambridge, MA 02141 (US)

(72) Inventors:
  • RUAN, Guoxiang
    Bridgewater, NJ 08807 (US)
  • SCARIA, Abraham
    Bridgewater, NJ 08807 (US)

(74) Representative: Mathys & Squire 
The Shard 32 London Bridge Street
London SE1 9SG
London SE1 9SG (GB)


(56) References cited: : 
WO-A1-2014/204724
 WO-A1-2015/070083
   
  • Levy: "Molecular Therapy Volume 23, Supplement 1, May 2015", , 27 April 2015 (2015-04-27), XP55287857, Retrieved from the Internet: URL:http://www.nature.com/mt/journal/v23/n 1s/pdf/mt201574a.pdf [retrieved on 2016-07-12] -& "ASGCT 18th Annual Meeting", American Soceity of Gene and Cell Therapy , 27 April 2015 (2015-04-27), XP002759938, Retrieved from the Internet: URL:http://www.asgct.org/meetings-educatio nal-programs/asgct-annual-meetings/archive d-annual-meetings/2015-annual-meeting [retrieved on 2016-07-14]
  • Dominique L Ouellet: "Deletion of GAA Repeats Expansion from the Intron 1 of the Frataxin Gene Using CRISPR/Cas9 System", , 13 May 2015 (2015-05-13), XP55288473, Retrieved from the Internet: URL:http://www.abstracts2view.com/asgct/vi ew.php?nu=ASGCT15L1_128 [retrieved on 2016-07-14]
  • Anonymous: "Addgene: CRISPR in the Lab: A Practical Guide", , 14 May 2015 (2015-05-14), XP55288205, Retrieved from the Internet: URL:https://web.archive.org/web/2015051404 5603/http://www.addgene.org/crispr/guide/ [retrieved on 2016-07-13]
  • ANN RAN F ET AL: "Genome engineering using the CRISPR-Cas9 system", NATURE PROTOCOLS, NATURE PUBLISHING GROUP, GB, vol. 8, no. 11, November 2013 (2013-11), pages 2281-2308, XP002744123, ISSN: 1754-2189, DOI: 10.1038/NPROT.2013.143 [retrieved on 2013-10-24]
  • MOORE ET AL: "CRISPR- based self-cleaving mechanism for controllable gene delivery in human cells", NUCLEIC ACIDS RESEARCH,, vol. 43, 1 January 2015 (2015-01-01), pages 1297-1303, XP002761477,
   
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