Patent SG-11201808920R-A (SG)
Title: Genome editing of human neural stem cells using nucleases
Inventor: PORTEUS MATTHEW
IPC: C12N15/90
Filed: 2017-04-13
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 11111111011110111010101111101011111011101111011011101011111011111011110111111 International Bureau .. .... ..Yejd ..... .....!;,,, (10) International Publication Number (43) International Publication Date WO 2017/180926 Al 19 October 2017(19.10.2017) WIPO I PCT (51) International Patent Classification: C12N 15/09 (2006.01) A61K C12N 15/90 (2006.01) A61K C12N 5/0797 (2006.01) (21) International Application Number: (22) International Filing Date: (25) Filing Language: (26) Publication Language: (30) Priority Data: 62/322,652 14 April 2016 (71) Applicants: BOCO SILICON 5201 Great America Parkway, 95054 (US). THE BOARD LELAND STANFORD JUNIOR [US/US]; Office of the General Counsel, Building 170, 3rd Floor, Main Quad, P.O. Box 20386, 2038 (US). Trustees of the Leland Stanford Junior University, Office 48/00 (2006.01) of the General Counsel, Building 170, 3rd Floor, Main 35/30 (2015.01) Quad, P.O. Box 20386, Stanford, CA 94305-2038 (US). TSUKAMOTO, Ann; c/o Boco Silicon Valley, Inc., 5201 Great America Parkway, Suite 320, Santa Clara, CA 95054 (US). UCHIDA, Nobuko; c/o Boco Silicon Valley, Inc., PCT/US2017/027504 5201 Great America Parkway, Suite 320, Santa Clara, CA 95054 (US). 13 April 2017 (13.04.2017) (74) Agents: MAO, Jian et al.; Morrisson & Foerster LLP, 755 English Page Mill Road, Palo Alto, CA 94304-1018 (US). English (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (14.04.2016) US BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, VALLEY. INC. [US/US]; DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, Suite 320, Santa Clara, CA HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, OF TRUSTEES OF THE KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, UNIVERSITY MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, Stanford, CA 94305- RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. H.; c/o The Board of Junior University, Office (84) Designated States (unless otherwise indicated, for every 170, 3rd Floor, Main kind of regional protection available): ARIPO (BW, GH, CA 94305-2038 (US). GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, Board of Trustees of the TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, Office of the General TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, Main Quad, P.O. Box DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, L (US). DEVER, Daniel, V, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, the Leland Stanford Junior SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, Counsel, Building 170, GW, KM, ML, MR, NE, SN, TD, TG). Box 20386, Stanford, CA Published: T.; c/o The Board of — with international search report (Art. 21(3)) (72) Inventors: PORTEUS, Matthew, Trustees of the Leland Stanford the General Counsel, Building of Quad, P.O. Box 20386, Stanford, KILDEBECK, Eric, J.; c/o The Leland Stanford Junior University, Counsel, Building 170, 3rd Floor, 20386, Stanford, CA 94305-2038 c/o of of P.; The Board Trustees University, Office of the General 3rd Floor, Main Quad, P.O. 94305-2038 (US). CLARK, Joseph, HUMAN NEURAL STEM CELLS USING NUCLEASES Title: GENOME EDITING OF (54) UN Locus HR Donor R 7 1---ir II 1 iiiagi . 1-1 Targeted Intearation ,tD ei tz. © GC 1-1 IN 1-1 (57) : The invention provides N human neural stem cells, and pharmaceutical compositions comprising the genetically modified human neural stem cells. In particu- lar said methods involve the use of a DNA nuclease, such as TALEN, ZFN or CRISPR/Cas mediated genome editing. Also provided C are associated kits. The invention also provides methods for preventing or treating a neurodegenerative disease or a neurological in - jury in a human subject using genetically modified human neural stem cells. - 911bp FIG. 16 methods for generating a genetically modified human neural stem cell, genetically modified