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1. WO2020142479 - DUX4 RNA SILENCING USING RNA TARGETING CRISPR-CAS13B

Publication Number WO/2020/142479
Publication Date 09.07.2020
International Application No. PCT/US2019/069048
International Filing Date 31.12.2019
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
CPC
C12N 15/113
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; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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]
Applicants
  • RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL [US]/[US]
Inventors
  • HARPER, Scott, Quenton
  • RASHNONEJAD, Afrooz
Agents
  • JANULIS, Lynn, L.
Priority Data
62/786,67031.12.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) DUX4 RNA SILENCING USING RNA TARGETING CRISPR-CAS13B
(FR) EXTINCTION D'ARN DUX4 À L'AIDE D'ARN CIBLANT CRISPR-CAS13B
Abstract
(EN)
RNA interference-based products and methods for inhibiting the expression of the double homeobox 4 (DUX4) gene on human chromosome 4q35 are disclosed. The disclosure includes the Casl3 protein silencing of RNA, wherein Casl3 is specifically targeted to a DUX4 region of interest using a sequence- specific guide RNA (gRNA). Recombinant adeno-associated viruses of the disclosure deliver DNAs encoding inhibitory gRNAs that are constructed with Casl3 direct repeats to knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD), and other disorders associated with elevated DUX4 expression, including cancer.
(FR)
L'invention concerne des produits à base d'interférence ARN et Des procédés d'inhibition de l'expression du gène double homéoboîte 4 (DUX4) sur le chromosome humain 4q35. L'invention comprend le silençage de Protéine Casl3 De l'ARN, où Casl3 est spécifiquement Ciblée sur une région DUX4 d'intérêt à l'Aide d'un ARN guide spécifique à une séquence (gARN). Les virus adéno-associés recombinants de l'invention Délivrent des ADN Codant pour des arng inhibiteurs qui sont construits Avec des répétitions directes Casl3 pour inactiver l'expression du DUX4. Les procédés ont une application dans le traitement de dystrophies musculaires comprenant, mais sans y être limitées, la dystrophie musculaire facio-scapulo-Humérale (FSHD), et d'autres troubles associés à une expression de DUX4 élevée, y compris le cancer.
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