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1. WO2021067788 - CRISPR SYSTEMS WITH ENGINEERED DUAL GUIDE NUCLEIC ACIDS

Publication Number WO/2021/067788
Publication Date 08.04.2021
International Application No. PCT/US2020/054050
International Filing Date 02.10.2020
IPC
C12N 15/113 2010.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/713 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
713Double-stranded nucleic acids or oligonucleotides
CPC
C12N 15/111
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
111General methods applicable to biologically active non-coding nucleic acids
C12N 2310/20
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
C12N 2310/315
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2310Structure or type of the nucleic acid
30Chemical structure
31of the backbone
315Phosphorothioates
C12N 2310/533
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2310Structure or type of the nucleic acid
50Physical structure
53partially self-complementary or closed
533having a mismatch or nick in at least one of the strands
Applicants
  • ARTISAN DEVELOPMENT LABS, INC. [US]/[US]
  • DANMARKS TEKNISKE UNIVERSITET [DK]/[DK]
Inventors
  • GILL, Ryan, T.
  • WARNECKE, Tanya
  • BARGHETTI, Andrea
  • POULSEN, Line, Dahl
Agents
  • XU, Lily
  • BRODOWSKI, Michael, H.
  • ASHRAF, Shovon
  • ARGENTIERI, Steven, R.
  • BIEKER, Kristina
Priority Data
62/910,05503.10.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) CRISPR SYSTEMS WITH ENGINEERED DUAL GUIDE NUCLEIC ACIDS
(FR) SYSTÈMES DE CRISPR AVEC ACIDES NUCLÉIQUES À DOUBLE GUIDE MODIFIÉS
Abstract
(EN)
The present invention relates to an engineered Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system comprising engineered dual guide nucleic acids (e.g., RNAs) capable of activating a CRISPR- Associated (Cas) nuclease, such as a type V-A Cas nuclease. Also provided are methods of targeting, editing, and/or modifying a nucleic acid using the engineered CRISPR system, and compositions and cells comprising the engineered CRISPR system.
(FR)
La présente invention concerne un système de répétitions palindromiques courtes groupées et régulièrement espacées (CRISPR) modifié comprenant des acides nucléiques à double guide modifiés (par exemple, des ARN) pouvant activer une nucléase associée à des CRISPR (Cas), telle qu'une nucléase Cas de type V-A. L'invention concerne également des méthodes de ciblage, d'édition et/ou de modification d'un acide nucléique à l'aide du système de CRISPR modifié, ainsi que des compositions et des cellules comprenant le système de CRISPR modifié.
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