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<ep-patent-document id="EP18815370B8W1" file="EP18815370W1B8.xml" lang="en" country="EP" doc-number="3701028" kind="B8" correction-code="W1" date-publ="20241023" status="c" dtd-version="ep-patent-document-v1-7">
<SDOBI lang="en"><B000><eptags><B001EP>ATBECHDEDKESFRGBGRITLILUNLSEMCPTIESILTLVFIROMKCYALTRBGCZEEHUPLSK..HRIS..MTNORS..SM..................</B001EP><B003EP>*</B003EP><B005EP>J</B005EP><B007EP>0009290-CORR01</B007EP></eptags></B000><B100><B110>3701028</B110><B120><B121>CORRECTED EUROPEAN PATENT SPECIFICATION</B121></B120><B130>B8</B130><B132EP>B1</B132EP><B140><date>20241023</date></B140><B150><B151>W1</B151><B153>73</B153><B155><B1551>de</B1551><B1552>Bibliographie</B1552><B1551>en</B1551><B1552>Bibliography</B1552><B1551>fr</B1551><B1552>Bibliographie</B1552></B155></B150><B190>EP</B190></B100><B200><B210>18815370.4</B210><B220><date>20181024</date></B220><B240><B241><date>20200522</date></B241><B242><date>20210526</date></B242></B240><B250>en</B250><B251EP>en</B251EP><B260>en</B260></B200><B300><B310>201762576277 P</B310><B320><date>20171024</date></B320><B330><ctry>US</ctry></B330><B310>201862664800 P</B310><B320><date>20180430</date></B320><B330><ctry>US</ctry></B330><B310>201862673039 P</B310><B320><date>20180517</date></B320><B330><ctry>US</ctry></B330><B310>201862690284 P</B310><B320><date>20180626</date></B320><B330><ctry>US</ctry></B330><B310>201862746900 P</B310><B320><date>20181017</date></B320><B330><ctry>US</ctry></B330></B300><B400><B405><date>20241023</date><bnum>202443</bnum></B405><B430><date>20200902</date><bnum>202036</bnum></B430><B450><date>20240911</date><bnum>202437</bnum></B450><B452EP><date>20240404</date></B452EP><B480><date>20241023</date><bnum>202443</bnum></B480></B400><B500><B510EP><classification-ipcr sequence="1"><text>C12N  15/113       20100101AFI20190503BHEP        </text></classification-ipcr><classification-ipcr sequence="2"><text>C12N  15/90        20060101ALI20190503BHEP        </text></classification-ipcr><classification-ipcr sequence="3"><text>C12N   9/22        20060101ALI20190503BHEP        </text></classification-ipcr><classification-ipcr sequence="4"><text>A61P  37/02        20060101ALI20190503BHEP        </text></classification-ipcr></B510EP><B520EP><classifications-cpc><classification-cpc sequence="1"><text>A61K  48/005       20130101 LI20190411BHEP        </text></classification-cpc><classification-cpc sequence="2"><text>C12N   9/22        20130101 LI20190211BHEP        </text></classification-cpc><classification-cpc sequence="3"><text>C12N  15/113       20130101 FI20190211BHEP        </text></classification-cpc><classification-cpc sequence="4"><text>C12N  15/907       20130101 LI20190211BHEP        </text></classification-cpc><classification-cpc sequence="5"><text>C12N2750/14143     20130101 LA20190411BHEP        </text></classification-cpc><classification-cpc sequence="6"><text>C12N2310/20        20170501 LA20190211BHEP        </text></classification-cpc><classification-cpc sequence="7"><text>A61P  37/02        20180101 LI20190527BHEP        </text></classification-cpc><classification-cpc sequence="8"><text>A61K  39/461       20230501 LI20240322RHEP        </text></classification-cpc><classification-cpc sequence="9"><text>A61K  39/4611      20230501 LI20240322RHEP        </text></classification-cpc><classification-cpc sequence="10"><text>A61K  39/464411    20230501 LI20240322RHEP        </text></classification-cpc></classifications-cpc></B520EP><B540><B541>de</B541><B542>SYSTEME UND VERFAHREN ZUR BEHANDLUNG VON HYPER-IGM-SYNDROM</B542><B541>en</B541><B542>SYSTEMS AND METHODS FOR TREATING HYPER-IGM SYNDROME</B542><B541>fr</B541><B542>SYSTÈMES ET MÉTHODES DE TRAITEMENT DU SYNDROME D'HYPER-IGM</B542></B540><B560><B561><text>WO-A1-2016/183345</text></B561><B562><text>TAHARA MINORU ET AL: "Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency", NATURE MEDICINE, vol. 10, no. 8, 25 July 2004 (2004-07-25), New York, pages 835 - 841, XP055805708, ISSN: 1078-8956, Retrieved from the Internet &lt;URL:https://www.nature.com/articles/nm1086.pdf&gt; DOI: 10.1038/nm1086</text></B562><B562><text>TAHARA  MINORU ET AL: "Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency - Supplementary Methods", NATURE MEDICINE, 25 July 2004 (2004-07-25), XP055805730, Retrieved from the Internet &lt;URL:https://static-content.springer.com/esm/art:10.1038/nm1086/MediaObjects/41591_2004_BFnm1086_MOESM4_ESM.pdf&gt; [retrieved on 20210519]</text></B562><B562><text>CAROLINE KUO: "Targeted Gene Therapy in the Treatment of X-Linked Hyper IgM Syndrome", CDI SYMPOSIUM 2016, May 2016 (2016-05-01), XP055554527, Retrieved from the Internet &lt;URL:https://www.uclahealth.org/mattel/cdi/workfiles/Symposium/2016/CKuo-CDI-Symposium-2016.pdf&gt; [retrieved on 20190211]</text></B562><B562><text>HUBBARD NICHOLAS ET AL: "Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome", BLOOD, vol. 127, no. 21, 22 February 2016 (2016-02-22), US, pages 2513 - 2522, XP055397792, ISSN: 0006-4971, DOI: 10.1182/blood-2015-11-683235</text></B562><B562><text>NICHOLAS HUBBARD ET AL: "Supplemental Methods", BLOOD, 22 February 2016 (2016-02-22), XP055554515, Retrieved from the Internet &lt;URL:http://www.bloodjournal.org/content/bloodjournal/suppl/2016/02/22/blood-2015-11-683235.DC1/blood-2015-11-683235-1.pdf&gt; 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KUO ET AL: "121. Targeted Gene Therapy in the Treatment of X-Linked Hyper IgM Syndrome", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY, vol. 23, May 2015 (2015-05-01), US, pages S50, XP055554579, ISSN: 1525-0016, DOI: 10.1016/S1525-0016(16)33726-1</text></B562><B562><text>CAROLINE Y. KUO ET AL: "Site Specific Gene Correction of Defects in CD40 Ligand Using the Crispr/Cas9 Genome Editing Platform", JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, vol. 135, no. 2, 2 February 2015 (2015-02-02), AMSTERDAM, NL, pages AB17, XP055554584, ISSN: 0091-6749, DOI: 10.1016/j.jaci.2014.12.987</text></B562><B562><text>CAROLINE Y. KUO ET AL: "Patient Specific Targeted Gene Therapy In The Treatment Of X-Linked Hyper-IgM Syndrome", JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, vol. 133, no. 2, 23 January 2014 (2014-01-23), AMSTERDAM, NL, pages AB162, XP055554585, ISSN: 0091-6749, DOI: 10.1016/j.jaci.2013.12.588</text></B562><B562><text>MATTHEW H. PORTEUS: "Knock-in editing: it functionally corrects!", BLOOD, vol. 127, no. 21, 26 May 2016 (2016-05-26), US, pages 2507 - 2509, XP055554520, ISSN: 0006-4971, DOI: 10.1182/blood-2016-03-703181</text></B562><B562><text>A. JAIN ET AL: "Partial immune reconstitution of X-linked hyper IgM syndrome with recombinant CD40 ligand", BLOOD, vol. 118, no. 14, 12 August 2011 (2011-08-12), US, pages 3811 - 3817, XP055554490, ISSN: 0006-4971, DOI: 10.1182/blood-2011-04-351254</text></B562><B562><text>JULIETTE M. K. M. DELHOVE ET AL: "Genome-Edited T Cell Therapies", CURRENT STEM CELL REPORTS, vol. 3, no. 2, 18 April 2017 (2017-04-18), pages 124 - 136, XP055554337, DOI: 10.1007/s40778-017-0077-5</text></B562><B562><text>CAROLINE Y. KUO ET AL: "Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome", CELL REPORTS, vol. 23, no. 9, 29 May 2018 (2018-05-29), US, pages 2606 - 2616, XP055554129, ISSN: 2211-1247, DOI: 10.1016/j.celrep.2018.04.103</text></B562></B560></B500><B700><B720><B721><snm>COTTA-RAMUSINO, Cecillia</snm><adr><str>36 Prince Street
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