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1. (WO2019005856) CRISPR LOGIC CIRCUITS FOR SAFER AND CONTROLLABLE GENE THERAPIES
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Pub. No.: WO/2019/005856 International Application No.: PCT/US2018/039583
Publication Date: 03.01.2019 International Filing Date: 26.06.2018
IPC:
A61K 48/00 (2006.01) ,C12N 15/00 (2006.01) ,C12N 15/87 (2006.01) ,C07H 21/04 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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
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
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21
Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
04
with deoxyribosyl as saccharide radical
Applicants:
KIANI, Samira [IR/US]; US
PRADHAN, Swechchha [NP/US]; US
MOGHADAM, Farzaneh [IR/US]; US
EBRAHIMKHANI, Mohammad Reza [IR/US]; US
ARIZONA BOARD OF REGENTS ON BEHALF OF ARIZONA STATE UNIVERSITY [US/US]; 1475 North Scottsdale Road, Suite 200 Scottsdale, AZ 85257, US
Inventors:
KIANI, Samira; US
PRADHAN, Swechchha; US
MOGHADAM, Farzaneh; US
EBRAHIMKHANI, Mohammad Reza; US
Agent:
LEWIS, Jessica L.; US
Priority Data:
62/524,95626.06.2017US
62/552,32130.08.2017US
Title (EN) CRISPR LOGIC CIRCUITS FOR SAFER AND CONTROLLABLE GENE THERAPIES
(FR) CIRCUITS LOGIQUES CRISPR POUR THÉRAPIES GÉNIQUES PLUS SÛRES ET CONTRÔLABLES
Abstract:
(EN) Aspects of the disclosure relate to synthetic regulatory systems composed of a multifunctional Cas nuclease, at least two guide RNAs (gRNAs) configured to target distinct nucleotide sequences, and a multilayered regulatory control element comprising ribozyme-based safety switches providing spatial and temporal control over the synthetic circuit in vivo.
(FR) Des aspects de l'invention concernent des systèmes de régulation synthétiques composés d'une nucléase Cas multifonctionnelle, d'au moins deux ARN de guidage (ARNg) configurés pour cibler des séquences nucléotidiques distinctes, et un élément de contrôle de régulation multicouche comprenant des commutateurs de sécurité à base de ribozyme assurant un contrôle spatial et temporel sur le circuit de synthèse in vivo.
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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)