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1. WO2021072361 - GENE EDITING TO CORRECT ANEUPLOIDIES AND FRAME SHIFT MUTATIONS

Publication Number WO/2021/072361
Publication Date 15.04.2021
International Application No. PCT/US2020/055223
International Filing Date 12.10.2020
IPC
C12N 9/22 2006.1
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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 15/09 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
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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 15/113 2010.1
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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
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/85 2006.1
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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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
C12N 15/90 2006.1
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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
A61K 48/005
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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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
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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
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
C12N 15/907
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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
907in mammalian cells
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2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
C12N 2320/34
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2320Applications; Uses
30Special therapeutic applications
34Allele or polymorphism specific uses
Applicants
  • THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK [US]/[US]
Inventors
  • EGLI, Dietrich
  • TREFF, Nathan
Agents
  • BONNIE KRAMER CARNEY
Priority Data
62/913,64710.10.2019US
63/011,42517.04.2020US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) GENE EDITING TO CORRECT ANEUPLOIDIES AND FRAME SHIFT MUTATIONS
(FR) ÉDITION DE GÈNE DESTINÉE À CORRIGER DES ANEUPLOÏDIES ET DES MUTATIONS DE MODIFICATION DE PHASE
Abstract
(EN)
The present disclosure relates to using CRISPR-based methods to perform gene editing to correct frame shift mutations in alleles with detectable phenotypes, and to correct aneuploidies. In one embodiment, the disclosure provides for methods for correcting an aneuploidy in an embryo comprising Introducing into the embryo: (i) at least one guide RNA (gRNA) or DNA encoding at least one guide RNA (gRNA); and (ii) at least one RNA-guided endonuclease or DNA encoding an RNA-guided endonuclease, wherein the endonuclease introduces at least one double-stranded break in a targeted site resulting in the loss or elimination of the extra chromosome.
(FR)
La présente divulgation concerne l'utilisation de procédés basés sur des CRISPR permettant d'effectuer une édition de gène destinée à corriger des mutations de modification de phase dans des allèles avec des phénotypes détectables, et destinée à corriger des aneuploïdies. Dans un mode de réalisation, l'invention concerne des procédés de correction d'une aneuploïdie dans un embryon comprenant l'introduction dans l'embryon : (i) d'au moins un ARN guide (ARNg) ou d'ADN codant pour au moins un ARN guide (ARNg); et (ii) d'au moins une endonucléase guidée par ARN ou d'un ADN codant pour une endonucléase guidée par ARN, l'endonucléase introduisant au moins une cassure double brin dans un site ciblé conduisant à la perte ou à l'élimination du chromosome supplémentaire.
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