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1. WO2020186213 - NOVEL NUCLEIC ACID MODIFIERS

Publication Number WO/2020/186213
Publication Date 17.09.2020
International Application No. PCT/US2020/022756
International Filing Date 13.03.2020
IPC
C12N 9/22 2006.1
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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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
C12N 15/11 2006.1
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
C12N 15/90 2006.1
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
CPC
C12N 15/102
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
10Processes for the isolation, preparation or purification of DNA or RNA
102Mutagenizing nucleic acids
C12N 15/111
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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 15/90
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
C12N 15/902
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
902using homologous recombination
C12N 2310/20
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2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
C12N 2310/3181
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2310Structure or type of the nucleic acid
30Chemical structure
31of the backbone
318where the PO2 is completely replaced, e.g. MMI or formacetal
3181Peptide nucleic acid, PNA
Applicants
  • THE BROAD INSTITUTE, INC. [US]/[US]
  • THE BRIGHAM AND WOMEN'S HOSPITAL, INC. [US]/[US]
Inventors
  • CHOUDHARY, Amit
  • VEDAGOPURAM, Sreekanth
  • SHOBA, Veronika
Agents
  • RUTLEDGE, Rachel D.
  • NIX, F. Brent
Priority Data
62/818,63114.03.2019US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) NOVEL NUCLEIC ACID MODIFIERS
(FR) NOUVEAUX MODIFICATEURS D'ACIDE NUCLÉIQUE
Abstract
(EN) The present inventions generally relate to site-specific delivery of nucleic acid modifiers and includes novel DNA-binding proteins and effectors that can be rapidly programmed to make site-specific DNA modifications. The present inventions also provide a synthetic all-in-one genome editor (SAGE) systems comprising designer DNA sequence readers and a set of small molecules that induce double-strand breaks, enhance cellular permeability, inhibit NHEJ and activate HDR, as well as methods of using and delivering such systems.
(FR) La présente invention concerne d'une manière générale l'administration spécifique à un site de modificateurs d'acide nucléique et comprend de nouvelles protéines de liaison à l'ADN et des effecteurs qui peuvent être rapidement programmés pour mettre en oeuvre des modifications d'ADN spécifiques à un site. La présente invention concerne également des systèmes éditeurs de génome synthétique tout-en-un (SAGE) des systèmes comprenant des lecteurs de séquence d'ADN de concepteur et un ensemble de petites molécules qui induisent des ruptures double brin, améliorent la perméabilité cellulaire, inhibent la NHEJ et activent les HDR, ainsi que des procédés d'utilisation et d'administration de tels systèmes.
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