ATBECHDEDKESFRGBGRITLILUNLSEMCPTIESILTLVFIROMKCYALTRBGCZEEHUPLSKBAHRIS..MTNORSMESM..................*J2.0.21 - 2999001/03298134CORRECTED EUROPEAN PATENT SPECIFICATIONB8B120230621W173deBibliographieenBibliographyfrBibliographieEP16718840.8201604152017121320200219enenen201562162720 P20150516US2023062120232520180328201813202305102023192022120220230621202325C12N 9/22 20060101AFI20171214BHEP C12N 15/00 20060101ALI20171214BHEP A61P 27/00 20060101ALI20171214BHEP A61P 21/00 20060101ALI20171214BHEP A61P 25/00 20060101ALI20171214BHEP A61P 3/00 20060101ALI20171214BHEP A61K 31/7105 20130101 LI20170620BHEP A61K2300/00 20130101 LI20170620BHEP A61K 31/7088 20130101 LI20170620BHEP A61K2300/00 20130101 LI20170620BHEP A61K 38/465 20130101 LI20170929BHEP A61K2300/00 20130101 LI20170929BHEP A61K 31/7088 20130101 LI20170620BHEP A61K 31/7105 20130101 LI20170620BHEP C12N2750/14143 20130101 LA20180329BHEP A61K 38/465 20130101 FI20170612BHEP C12N 9/22 20130101 LI20190124BHEP A61P 11/00 20180101 LI20200318BHEP A61P 13/02 20180101 LI20200319BHEP A61P 13/12 20180101 LI20200319BHEP A61P 21/00 20180101 LI20200320BHEP A61P 21/02 20180101 LI20200320BHEP A61P 21/04 20180101 LI20200320BHEP A61P 25/00 20180101 LI20200320BHEP A61P 25/02 20180101 LI20200323BHEP A61P 25/28 20180101 LI20200324BHEP A61P 27/00 20180101 LI20200324BHEP A61P 27/02 20180101 LI20200324BHEP A61P 27/12 20180101 LI20200324BHEP A61P 3/00 20180101 LI20200326BHEP A61P 3/12 20180101 LI20200327BHEP A61P 35/00 20180101 LI20200327BHEP A61P 35/02 20180101 LI20200327BHEP A61P 3/08 20180101 LI20200330BHEP A61P 37/02 20180101 LI20200331BHEP A61P 43/00 20180101 LI20200331BHEP A61P 5/00 20180101 LI20200331BHEP A61P 7/02 20180101 LI20200331BHEP A61P 7/04 20180101 LI20200331BHEP A61P 7/06 20180101 LI20200331BHEP A61P 9/00 20180101 LI20200331BHEP deGEN-EDITING VON TIEFEN INTRONISCHEN MUTATIONENenGENE EDITING OF DEEP INTRONIC MUTATIONSfrÉDITION GÉNIQUE DE MUTATIONS INTRONIQUES PROFONDESWO-A1-2014/204724WO-A1-2015/070083Levy: "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,RUAN, Guoxiangc/o Sanofi 55 Corporate Drive Mail Code 55A-505ABridgewater, NJ 08807USSCARIA, Abrahamc/o Sanofi 55 Corporate Drive Mail Code 55A-505ABridgewater, NJ 08807USGenzyme Corporation101954794P50738EP-PCT450 Water StreetCambridge, MA 02141USMathys & Squire100061165The Shard 32 London Bridge StreetLondon SE1 9SGGBALATBEBGCHCYCZDEDKEEESFIFRGBGRHRHUIEISITLILTLULVMCMKMTNLNOPLPTRORSSESISKSMTRBA20171213ME20171213US201602798720160415enWO20161867722016112420164720170105000000