JP2024515827A - 多重ＣＲＩＳＰＲ／Ｃａｓ９媒介標的遺伝子活性化システム - Google Patents

多重ＣＲＩＳＰＲ／Ｃａｓ９媒介標的遺伝子活性化システム Download PDF

Info

Publication number
JP2024515827A
JP2024515827A JP2023566512A JP2023566512A JP2024515827A JP 2024515827 A JP2024515827 A JP 2024515827A JP 2023566512 A JP2023566512 A JP 2023566512A JP 2023566512 A JP2023566512 A JP 2023566512A JP 2024515827 A JP2024515827 A JP 2024515827A
Authority
JP
Japan
Prior art keywords
nucleic acid
seq
sequence
modified
promoter
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023566512A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024515827A5 (https=
Inventor
ベルモンテ， ファン カルロス イズピスア
チャオ ワン，
シン－カイ リャオ，
プラディープ レディ，
Original Assignee
ソーク インスティテュート フォー バイオロジカル スタディーズ
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ソーク インスティテュート フォー バイオロジカル スタディーズ filed Critical ソーク インスティテュート フォー バイオロジカル スタディーズ
Publication of JP2024515827A publication Critical patent/JP2024515827A/ja
Publication of JP2024515827A5 publication Critical patent/JP2024515827A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—Aptamers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
  • Peptides Or Proteins (AREA)
JP2023566512A 2021-04-28 2022-04-28 多重ＣＲＩＳＰＲ／Ｃａｓ９媒介標的遺伝子活性化システム Pending JP2024515827A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163181059P 2021-04-28 2021-04-28
US63/181,059 2021-04-28
PCT/US2022/026805 WO2022232442A2 (en) 2021-04-28 2022-04-28 Multiplex crispr/cas9-mediated target gene activation system

Publications (2)

Publication Number Publication Date
JP2024515827A true JP2024515827A (ja) 2024-04-10
JP2024515827A5 JP2024515827A5 (https=) 2025-05-20

Family

ID=83848892

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023566512A Pending JP2024515827A (ja) 2021-04-28 2022-04-28 多重ＣＲＩＳＰＲ／Ｃａｓ９媒介標的遺伝子活性化システム

Country Status (7)

Country Link
US (1) US20240209354A1 (https=)
EP (1) EP4330375A4 (https=)
JP (1) JP2024515827A (https=)
CN (1) CN117580941A (https=)
AU (1) AU2022267320A1 (https=)
CA (1) CA3218209A1 (https=)
WO (1) WO2022232442A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025111340A1 (en) * 2023-11-20 2025-05-30 William Marsh Rice University Engineered extrachromosomal nucleic acids and methods of use thereof
WO2025227064A1 (en) * 2024-04-26 2025-10-30 Renasci Biotechnologies, Inc. Genomic editing methods to treat cardiovascular disease, and compositions for use in practicing the same
CN119020323A (zh) * 2024-09-29 2024-11-26 中国农业科学院作物科学研究所 一种融合外切酶的CRIPSR/Cas12i3小麦基因编辑体系

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017532001A (ja) * 2013-12-12 2017-11-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
JP2019504868A (ja) * 2016-02-15 2019-02-21 テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション レトロウイルス核酸配列の切除
WO2019236081A1 (en) * 2018-06-06 2019-12-12 Salk Institute For Biological Studies Targeted gene activation using modified guide rna
JP2021508451A (ja) * 2017-12-21 2021-03-11 クリスパー セラピューティクス アーゲー ２ａ型アッシャー症候群の処置のための材料および方法
US20210079394A1 (en) * 2019-09-13 2021-03-18 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017532001A (ja) * 2013-12-12 2017-11-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
JP2019504868A (ja) * 2016-02-15 2019-02-21 テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション レトロウイルス核酸配列の切除
JP2021508451A (ja) * 2017-12-21 2021-03-11 クリスパー セラピューティクス アーゲー ２ａ型アッシャー症候群の処置のための材料および方法
WO2019236081A1 (en) * 2018-06-06 2019-12-12 Salk Institute For Biological Studies Targeted gene activation using modified guide rna
US20210079394A1 (en) * 2019-09-13 2021-03-18 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles

Also Published As

Publication number Publication date
WO2022232442A3 (en) 2022-12-15
AU2022267320A9 (en) 2023-11-30
EP4330375A2 (en) 2024-03-06
WO2022232442A2 (en) 2022-11-03
US20240209354A1 (en) 2024-06-27
AU2022267320A1 (en) 2023-11-23
CA3218209A1 (en) 2022-11-03
EP4330375A4 (en) 2025-08-13
CN117580941A (zh) 2024-02-20

Similar Documents

Publication Publication Date Title
US12024728B2 (en) Methods and compositions for modulating a genome
US20210017509A1 (en) Gene Editing for Autosomal Dominant Diseases
JP7832262B2 (ja) 肝特異的ウイルスプロモーター及びその使用方法
AU2020391518B2 (en) APOE gene therapy
KR20220004681A (ko) 신규 aav 캡시드 및 이를 포함하는 조성물
JP2021502058A (ja) Ｒｎａを編集するための組成物および方法
JP2024515827A (ja) 多重ＣＲＩＳＰＲ／Ｃａｓ９媒介標的遺伝子活性化システム
JP2018512125A (ja) 多重ベクターシステム及びその使用
CN113710693B (zh) Dna结合结构域反式激活因子及其用途
WO2019236081A1 (en) Targeted gene activation using modified guide rna
CA3175625A1 (en) Crispr-inhibition for facioscapulohumeral muscular dystrophy
US20230279405A1 (en) Dna-binding domain transactivators and uses thereof
IL311223A (en) Recruitment in trans of gene editing system components
WO2021138286A1 (en) Self-complementary aav delivery system for crispr/cas9
TW202444915A (zh) Serpina調節組合物及方法
WO2023024504A1 (en) Crispr-cas13 system for treating sod1-associated diseases
JP2024529294A (ja) ミオシン重鎖塩基編集のための組成物および方法
CA3218631A1 (en) Vector system
US20260115322A1 (en) Recombinant tert-encoding viral genomes and vectors
US20260027236A1 (en) Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2023025103A1 (en) Crispr-cas13 system for treating sod1-associated diseases
JP2024529279A (ja) Ｒｂｍ２０変異のゲノム編集
WO2025128661A1 (en) Compositions and methods for targeting astrocytes to treat neurological disorders
HK40106592A (zh) 年龄相关疾病和病症的基因治疗方法
WO2025019820A1 (en) All in one vectors for the treatment of facioscapulohumeral muscular dystrophy

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250428

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250428

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20260304