JP2024528540A - 遺伝子操作されたガイドｒｎａ及びポリヌクレオチド - Google Patents

遺伝子操作されたガイドｒｎａ及びポリヌクレオチド Download PDF

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JP2024528540A
JP2024528540A JP2023580744A JP2023580744A JP2024528540A JP 2024528540 A JP2024528540 A JP 2024528540A JP 2023580744 A JP2023580744 A JP 2023580744A JP 2023580744 A JP2023580744 A JP 2023580744A JP 2024528540 A JP2024528540 A JP 2024528540A
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rna
target
relative
guide
internal loop
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Japanese (ja)
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JP2024528540A5 (https=
JPWO2023278449A5 (https=
Inventor
リチャード トーマス サリバン，
ブライアン ジョン ブース，
エイドリアン ブリッグス，
イアニス サバ，
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シェイプ セラピューティクス インコーポレイテッド
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Publication of JP2024528540A5 publication Critical patent/JP2024528540A5/ja
Publication of JPWO2023278449A5 publication Critical patent/JPWO2023278449A5/ja
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    • 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
    • C12N15/63—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
    • 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
    • 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

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023580744A 2021-06-29 2022-06-28 遺伝子操作されたガイドｒｎａ及びポリヌクレオチド Pending JP2024528540A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202163216178P 2021-06-29 2021-06-29
US63/216,178 2021-06-29
US202163277701P 2021-11-10 2021-11-10
US63/277,701 2021-11-10
US202263303680P 2022-01-27 2022-01-27
US63/303,680 2022-01-27
US202263345059P 2022-05-24 2022-05-24
US63/345,059 2022-05-24
PCT/US2022/035320 WO2023278449A1 (en) 2021-06-29 2022-06-28 Engineered guide rnas and polynucleotides

Publications (3)

Publication Number Publication Date
JP2024528540A true JP2024528540A (ja) 2024-07-30
JP2024528540A5 JP2024528540A5 (https=) 2025-08-07
JPWO2023278449A5 JPWO2023278449A5 (https=) 2025-08-07

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JP2023580744A Pending JP2024528540A (ja) 2021-06-29 2022-06-28 遺伝子操作されたガイドｒｎａ及びポリヌクレオチド

Country Status (6)

Country Link
US (1) US20240271139A1 (https=)
EP (1) EP4363579A1 (https=)
JP (1) JP2024528540A (https=)
AU (1) AU2022301997A1 (https=)
CA (1) CA3223109A1 (https=)
WO (1) WO2023278449A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3236391A1 (en) * 2021-10-26 2023-05-04 Yiannis SAVVA Rna-editing compositions and methods of use
WO2024137993A2 (en) * 2022-12-23 2024-06-27 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
WO2026006483A1 (en) * 2024-06-25 2026-01-02 Shape Therapeutics Inc. Snca vectors and engineered guide rnas
WO2026006334A1 (en) * 2024-06-25 2026-01-02 Shape Therapeutics Inc. Dux4 vectors and engineered guide rnas

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021113270A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing
CA3177380A1 (en) * 2020-05-26 2021-12-02 Shape Therapeutics Inc. Compositions and methods for modifying target rnas
WO2022103852A1 (en) * 2020-11-11 2022-05-19 Shape Therapeutics Inc. Rna-editing compositions and methods of use
WO2022119975A2 (en) * 2020-12-01 2022-06-09 Shape Therapeutics Inc. High throughput rna-editing screening methods

Also Published As

Publication number Publication date
AU2022301997A1 (en) 2024-01-04
US20240271139A1 (en) 2024-08-15
WO2023278449A1 (en) 2023-01-05
CA3223109A1 (en) 2023-01-05
EP4363579A1 (en) 2024-05-08

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