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1. (WO2019005884) CRISPR/CAS-ADENINE DEAMINASE BASED COMPOSITIONS, SYSTEMS, AND METHODS FOR TARGETED NUCLEIC ACID EDITING
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Pub. No.: WO/2019/005884 International Application No.: PCT/US2018/039616
Publication Date: 03.01.2019 International Filing Date: 26.06.2018
IPC:
A61K 48/00 (2006.01) ,C12N 15/00 (2006.01) ,C12N 15/82 (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
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79
Vectors or expression systems specially adapted for eukaryotic hosts
82
for plant cells
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:
THE BROAD INSTITUTE, INC. [US/US]; 415 Main Street Cambridge, Massachusetts 02142, US
MASSACHUSETTS INSTITUTE OF TECHNOLOGY [US/US]; 77 Massachusetts Avenue Cambridge, Massachusetts 02139, US
PRESIDENT AND FELLOWS OF HARVARD COLLEGE [US/US]; 17 Quincy Street Cambridge, Massachusetts 02138, US
Inventors:
ZHANG, Feng; US
GOOTENBERG, Jonathan; US
COX, David Benjamin Turitz; US
ABUDAYYEH, Omar; US
KANNAN, Soumya; US
Agent:
NIX, F. Brent; US
JIANG, Mengxi; US
Priority Data:
62/525,18126.06.2017US
62/528,39103.07.2017US
62/534,01618.07.2017US
62/561,63821.09.2017US
62/568,30404.10.2017US
62/574,15818.10.2017US
62/591,18727.11.2017US
62/610,10522.12.2017US
Title (EN) CRISPR/CAS-ADENINE DEAMINASE BASED COMPOSITIONS, SYSTEMS, AND METHODS FOR TARGETED NUCLEIC ACID EDITING
(FR) COMPOSITIONS À BASE DE CRISPR/CAS-ADÉNINE DÉSAMINASE, SYSTÈMES ET PROCÉDÉS D'ÉDITION CIBLÉE D'ACIDES NUCLÉIQUES
Abstract:
(EN) The invention provides for systems, methods, and compositions for targeting and editing nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a RNA-targeting Cas13 protein, at least one guide molecule, and at least one adenosine deaminase protein or catalytic domain thereof.
(FR) L'invention concerne des systèmes, des procédés et des compositions destinés au ciblage et à l’édition d'acides nucléiques. L'invention concerne, en particulier, des systèmes de ciblage d'ADN non naturels ou génétiquement modifiés comprenant une protéine Cpfl de ciblage d'ADN, au moins une molécule de guidage, et au moins une protéine d'adénosine désaminase ou un domaine catalytique de cette dernière.
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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)