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1. (WO2019067011) PROGRAMMED MODULATION OF CRISPR/CAS9 ACTIVITY
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Pub. No.: WO/2019/067011 International Application No.: PCT/US2018/016231
Publication Date: 04.04.2019 International Filing Date: 31.01.2018
IPC:
C12N 9/22 (2006.01) ,C12N 15/90 (2006.01) ,A61K 38/46 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14
Hydrolases (3.)
16
acting on ester bonds (3.1)
22
Ribonucleases
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
87
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90
Stable introduction of foreign DNA into chromosome
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38
Medicinal preparations containing peptides
16
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43
Enzymes; Proenzymes; Derivatives thereof
46
Hydrolases (3)
Applicants:
KANSAS STATE UNIVERSITY RESEARCH FOUNDATION [US/US]; 2005 Research Park Circle, Suite 105 Manhattan, Kansas 66502, US
Inventors:
FINNIGAN, Gregory; US
Agent:
COOK, Crissa A.; US
Priority Data:
62/565,65129.09.2017US
Title (EN) PROGRAMMED MODULATION OF CRISPR/CAS9 ACTIVITY
(FR) MODULATION PROGRAMMÉE DE L'ACTIVITÉ CRISPR/CAS9
Abstract:
(EN) Modifications to the CRISPR-Cas gene editing and gene drive systems are described for inhibiting the effectiveness of the gene editing system, such as by including a regulatory element that reduces expression of the CRISPR nuclease in the cell, or by inducing one or more base pair mismatches in the single guide RNA to reduce specificity of the system to the target sequence, or by including one or more nuclear export signal sequences to reduce accumulation of the CRISPR nuclease in the nucleus of the cell, as well as by inducing competition between the CRISPR nuclease by inclusion of a secondary functional or dead CRISPR nuclease in the system. Anti- CRISPR peptides that inhibit the activity of the CRISPR nuclease are also described.
(FR) L'invention décrit des modifications apportées aux systèmes d'édition de gènes CRISPR-Cas et d'entraînement de gènes pour inhiber l'efficacité du système d'édition génique, par exemple par l'inclusion d'un élément régulateur qui réduit l'expression de la nucléase CRISPR dans la cellule ou par l'induction d'un ou de plusieurs mésappariements de paires de bases dans l'ARN guide unique pour réduire la spécificité du système vis-à-vis de la séquence cible ou par l'inclusion d'une ou de plusieurs séquences de signal d'exportation nucléaire pour réduire l'accumulation de la nucléase CRISPR dans le noyau de la cellule, ainsi que par l'induction d'une compétition entre la nucléase CRISPR par inclusion d'une nucléase CRISPR secondaire fonctionnelle ou morte dans le système. L'invention concerne également des peptides anti-CRISPR qui inhibent l'activité de la nucléase CRISPR.
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Designated States: 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
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (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)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)