Patent SG-11201900038Q-A (SG)
Title: Compositions and methods comprising improvements of crispr guide rnas using the h1 promoter
Inventor: JASKULA-RANGA VINOD
IPC: C12N15/85
Filed: 2017-07-05
Abstract: OO cc O C () INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 11 January 2018 (11.01.2018) iii01111110111010100111 1101001111011010111101100M#011 (10) International Publication Number WO 2018/009534 Al WIPO I PCT (51) International Patent Classification: C12N 15/113 (2010.01) A61K 38/46 C12N 9/16 (2006.01) A61K 48/00 C12N 15/63 (2006.01) A61P 35/00 C12N 15/85 (2006.01) (22) International Filing Date: 05 July 2017 (05.07.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/358,335 05 July 2016 (05.07.2016) US (71) Applicant: THE JOHNS HOPKINS UNIVERSITY [US/US]; 3400 North Charles Street, Baltimore, MD 21218 (US). (72) Inventors: JASKULA-RANGA, Vinod; 1201 West Mount Royal Avenue, Unit 506, Baltimore, MD 21217 (US). ZACK, Donald; 3 Ruby Field Ct., Baltimore, MD 21209 (US). WELSBIE, Derek, S.; 400 North Broadway, Smith Building, Room 3035, Baltimore, MD 21231 (US). (74) Agent: RUSSELL, Hathaway P. et al.; Foley Hoag LLP, Seaport West, 155 Seaport Boulevard, Boston, MA 02210-2600 (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, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, 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, 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. (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, ST, 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, KM, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3)) (2006.01) (2006.01) (2006.01) (21) International Application Number: PCT/US2017/040707 (54) Title: COMPOSITIONS AND METHODS COMPRISING IMPROVEMENTS OF CRISPR GUIDE RNAS USING THE H1 PROMOTER (57) : The presently disclosed subject matter provides compositions and methods comprising improvements of a CRISPR system (e.g. CRISPR associated (Cas) 9 (CRISPR-Cas9, non-Cas9 CRISPR systems). Such compositions may comprise modifications to the H1 promoter region, addition of 5'UTR modifications, different orthologous sequences of the H1 promoter, novel compact bidirectional promoter sequences with both pol II and pol III activity, addition of Kozak consensus sequences, termination sequences, addition of conditional pol II/pol III bidirectional promoter expression, addition of a donor template sequence for correcting mutations, or combinations thereof. Other aspects of the invention relate to modifications to Cas9 through post-transcriptional cell-cycle regulation fusions, engineered partial target sites such that the nuclease can bind without DNA cleavage, auto-regulation sites, and N-terminal modifications to modulate half-life.