Patent SG-11201407702X-A (SG)
Title: Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
Inventor: DOUDNA JENNIFER A
IPC: C12N15/11
Filed: 2013-03-15
Abstract: (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 28 November 2013 (28.11.2013) WIPOIPCT (10) International Publication Number WO 2013/176772 A1 (51) International Patent Classification: C12N15/11 (2006.01) C07K19/00 (2006.01) C12N15/63 (2006.01) C12N 5/10 (2006.01) (21) International Application Number: (22) International Filing Date: (25) Filing Language: (26) Publication Language: (30) Priority Data: 61/652,086 61/716,256 61/757,640 61/765,576 PCT/US2013/032589 15 March 2013 (15.03.2013) English English US US us us 25 May 2012 (25.05.2012) 19 October 2012 (19.10.2012) 28 January 2013 (28.01.2013) 15 February 2013 (15.02.2013) (71) Applicants: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA [US/US]; 1111 Franklin Street, 12th Floor, Oakand, California 94607 (US). UNIVERSITY OF VIENNA [AT/AT]; Universitatsring 1, A-1010 Vienna (AT). DOUDNA, Jennifer A. [US/US]; 164 Vicente Road, Berkeley, California 94705 (US). (72) Inventors; and (71) Applicants : JINEK, Martin [CZ/US]; 1846 Spruce Street, Berkeley, California 94709 (US). CHARPENTI- ER, Emmanuelle. CHYLINSKI, Krzysztof. DOUDNA CATE, James Harrison [US/US]; 164 Vicente Road, Berkeley, California (US). LIM, Wendell [—/US]; 149 Collins Street, San Francisco, California 94118 (US). QI, Lei [—/US]; 730 Kinkead Way #302, Albany, California 94706 (US). (74) Agent: BORDEN, Paula A.; BOZICEVIC, FIELD & FRANCIS LLP., 1900 University Avenue, Suite 200, East Palo Alto, California 94303 (US). (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, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IS, JP, KE, KG, KM, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LT, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, 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. (84) Designated States (unless otherwise indicated, for every kind of regional protection available)'. ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: — of inventorship (Rule 4.17(iv)) Published: — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a)) CJ l> l> i> i-H cn i-H o CJ o & (54) Title: METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIR- ECTED MODULATION OF TRANSCRIPTION (57) Abstract: The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or polypeptide associated with a the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multi­ cellular organisms.