Patent SG-11201908658T-A (SG)
Title: Nucleobase editors comprising nucleic acid programmable dna binding proteins
Inventor: LIU DAVID
IPC: C12N15/11
Filed: 2018-03-23
Abstract: D/N/A 11 D/N/A 12 D/N/A 13 D/N/A 14 D/N/A 15 D/N/A irrc .• ANN Aftititie 1-1 01 O O SO N 1-1 00 O 1-1 N O (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 27 September 2018 (27.09.2018) WIP0 I PCT Iiiimmounnotiolomolomiloollionomovom (10) International Publication Number WO 2018/176009 Al (51) International Patent Classification: C12N 15/11 (2006.01) C12N 15/10 (2006.01) (21) International Application Number: PCT/US2018/024208 (22) International Filing Date: 23 March 2018 (23.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/475,830 23 March 2017 (23.03.2017) US 62/490,587 26 April 2017 (26.04.2017) US 62/511,934 26 May 2017 (26.05.2017) US 62/551,951 30 August 2017 (30.08.2017) US (71) Applicant: PRESIDENT AND FELLOWS OF HAR- VARD COLLEGE [US/US]; 17 Quincy Street, Cam- bridge, MA 02138 (US). (72) Inventors: LIU, David, R.; 3 Whitman Circle, Lexington, MA 02420 (US). KOMOR, Alexis, Christine; 9135 Judi- cial Drive, #3419, San Diego, CA 92122 (US). CHEN, Li- wei; 86 Avon Street, Apt. 2, Somerville, MA 02143 (US). REES, Holly, A.; 34 Hawthorne Street, Somerville, MA 02144 (US). (74) Agent: HEBERT, Alan, M.; Wolf, Greenfield & Sacks, P.C., 600 Atlantic Avenue, Boston, MA 02210-2206 (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. (54) Title: NUCLEOBASE EDITORS COMPRISING NUCLEIC ACID PROGRAMMABLE DNA BINDING PROTEINS FIGURE 152 (57) : Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for the targeted editing of nucleic acids, including editing a single site within the genome of a cell or subject, e. g., within the human genome. In some embodiments, fusion proteins of nucleic acid programmable DNA binding proteins (napDNAbp), e. g., Cpf 1 or variants thereof, and nucleic acid editing proteins or protein domains, e. g., deaminase domains, are provided. In some embodiments, methods for targeted nucleic acid editing are provided. In some embodiments, reagents and kits for the generation of targeted nucleic acid editing proteins, e. g., fusion proteins of a napDNAbp (e. g., CasX, CasY, Cpfl, C2c1, C2c2, C2C3, and Argonaute) and nucleic acid editing proteins or domains, are provided. [Continued on next page] WO 2018/176009 Al IMEDIMOMMIONE1013010MINNIEHOMEMOIMIE (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)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) with sequence listing part of description (Rule 5.2(a))