AU2022293494A1 - Systems and methods for regulating aberrant gene expressions - Google Patents

Systems and methods for regulating aberrant gene expressions Download PDF

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Publication number
AU2022293494A1
AU2022293494A1 AU2022293494A AU2022293494A AU2022293494A1 AU 2022293494 A1 AU2022293494 A1 AU 2022293494A1 AU 2022293494 A AU2022293494 A AU 2022293494A AU 2022293494 A AU2022293494 A AU 2022293494A AU 2022293494 A1 AU2022293494 A1 AU 2022293494A1
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Australia
Prior art keywords
target gene
gene
heterologous
krab
cell
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Pending
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AU2022293494A
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English (en)
Inventor
Abhinav ADHIKARI
Siddaraju BOREGOWDA
Giovanni CAROSSO
Alexandra Sylvie Collin De L'hortet
Timothy Daley
Thomas Blair GAINOUS
Daniel O. HART
Tengyu KO
Yanxia Liu
Thao Nguyen Vu LUONG
Amber Ruth SALZMAN
Rosemarie Wenting TSOA
Xiao Yang
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Epicrispr Biotechnologies Inc
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Epicrispr Biotechnologies Inc
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Publication of AU2022293494A1 publication Critical patent/AU2022293494A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007—Methyltransferases (general) (2.1.1.)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Health & Medical Sciences (AREA)
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  • Analytical Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2022293494A 2021-06-17 2022-06-16 Systems and methods for regulating aberrant gene expressions Pending AU2022293494A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163211791P 2021-06-17 2021-06-17
US63/211,791 2021-06-17
PCT/US2022/033797 WO2022266324A1 (en) 2021-06-17 2022-06-16 Systems and methods for regulating aberrant gene expressions

Publications (1)

Publication Number Publication Date
AU2022293494A1 true AU2022293494A1 (en) 2024-01-04

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AU2022293494A Pending AU2022293494A1 (en) 2021-06-17 2022-06-16 Systems and methods for regulating aberrant gene expressions

Country Status (11)

Country Link
US (1) US20240216482A1 (https=)
EP (1) EP4355357A4 (https=)
JP (1) JP2024523394A (https=)
KR (1) KR20240032038A (https=)
CN (1) CN117940150A (https=)
AU (1) AU2022293494A1 (https=)
CA (1) CA3221874A1 (https=)
GB (1) GB2624791A (https=)
IL (1) IL309174A (https=)
MX (1) MX2023014750A (https=)
WO (1) WO2022266324A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3192654A1 (en) 2020-09-01 2022-03-10 The Board Of Trustees Of The Leland Stanford Junior Unversity Synthetic miniature crispr-cas (casmini) system for eukaryotic genome engineering
WO2023168242A1 (en) 2022-03-01 2023-09-07 Epicrispr Biotechnologies, Inc. Engineered nucleases, compositions, and methods of use thereof
AU2024235956A1 (en) * 2023-03-16 2025-09-18 Epicrispr Biotechnologies, Inc. Systems and methods for regulating aberrant gene expressions
CN119955797B (zh) * 2025-01-13 2026-01-23 江南大学 Kif5b基因在调控成肌细胞生理活性中的功能和应用
CN119954926B (zh) * 2025-03-11 2025-10-28 华南农业大学 Klf5转录因子在调控猪骨骼肌发育中的应用及方法

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190127713A1 (en) * 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
CA3074723A1 (en) * 2016-09-23 2018-03-29 University Of Massachusetts Silencing of dux4 by recombinant gene editing complexes
AU2019419494B2 (en) * 2018-12-31 2026-03-26 Research Institute At Nationwide Children's Hospital DUX4 RNA silencing using RNA targeting CRISPR-Cas13b
WO2022133062A1 (en) * 2020-12-16 2022-06-23 Epicrispr Biotechnologies, Inc. Systems and methods for engineering characteristics of a cell

Also Published As

Publication number Publication date
MX2023014750A (es) 2024-03-08
WO2022266324A1 (en) 2022-12-22
JP2024523394A (ja) 2024-06-28
IL309174A (en) 2024-02-01
EP4355357A1 (en) 2024-04-24
GB2624791A (en) 2024-05-29
KR20240032038A (ko) 2024-03-08
GB202319324D0 (en) 2024-01-31
EP4355357A4 (en) 2025-08-20
CA3221874A1 (en) 2022-12-22
US20240216482A1 (en) 2024-07-04
CN117940150A (zh) 2024-04-26

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