JP2025509262A - アンジェルマン症候群における父方由来ＵＢＥ３Ａ遺伝子発現を回復させるためのＳＮＯＲＤ１１５の位置を標的とするｓｈＲＮＡ - Google Patents

アンジェルマン症候群における父方由来ＵＢＥ３Ａ遺伝子発現を回復させるためのＳＮＯＲＤ１１５の位置を標的とするｓｈＲＮＡ Download PDF

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JP2025509262A
JP2025509262A JP2024553307A JP2024553307A JP2025509262A JP 2025509262 A JP2025509262 A JP 2025509262A JP 2024553307 A JP2024553307 A JP 2024553307A JP 2024553307 A JP2024553307 A JP 2024553307A JP 2025509262 A JP2025509262 A JP 2025509262A
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seq
polynucleotide
shrna
ube3a
expression vector
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Pending
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JP2024553307A
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JPWO2023172535A5 (https=
JP2025509262A5 (https=
Inventor
チェンバレン，ストーミー
ジャーメイン，ノエル
ペリノ，ピーター
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Ovid Therapeutics Inc
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Ovid Therapeutics Inc
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Publication of JP2025509262A5 publication Critical patent/JP2025509262A5/ja
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    • 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
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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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    • C12N15/00—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
    • C12N15/1137—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 against enzymes
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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
    • C12N15/63—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
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    • C12N15/86—Viral vectors
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • 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
    • 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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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2024553307A 2022-03-07 2023-03-07 アンジェルマン症候群における父方由来ＵＢＥ３Ａ遺伝子発現を回復させるためのＳＮＯＲＤ１１５の位置を標的とするｓｈＲＮＡ Pending JP2025509262A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263317155P 2022-03-07 2022-03-07
US63/317,155 2022-03-07
PCT/US2023/014680 WO2023172535A2 (en) 2022-03-07 2023-03-07 shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME

Publications (3)

Publication Number Publication Date
JP2025509262A true JP2025509262A (ja) 2025-04-11
JPWO2023172535A5 JPWO2023172535A5 (https=) 2026-03-16
JP2025509262A5 JP2025509262A5 (https=) 2026-03-16

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ID=87935834

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JP2024553307A Pending JP2025509262A (ja) 2022-03-07 2023-03-07 アンジェルマン症候群における父方由来ＵＢＥ３Ａ遺伝子発現を回復させるためのＳＮＯＲＤ１１５の位置を標的とするｓｈＲＮＡ

Country Status (10)

Country Link
US (1) US20230332150A1 (https=)
EP (1) EP4473116A2 (https=)
JP (1) JP2025509262A (https=)
KR (1) KR20240155345A (https=)
CN (1) CN119384504A (https=)
AU (1) AU2023231550A1 (https=)
CA (1) CA3259398A1 (https=)
IL (1) IL315359A (https=)
MX (1) MX2024010820A (https=)
WO (1) WO2023172535A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025101533A1 (en) * 2023-11-09 2025-05-15 Encoded Therapeutics, Inc. Methods and compositions for increasing expression of ube3a

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150329857A1 (en) * 2011-12-28 2015-11-19 Universite Pierre Et Marie Curie (Paris 6) Rna interference to activate stem cells
PT3717646T (pt) * 2017-12-01 2025-07-28 Texas A & M Univ Sys Tratamento antisense da síndrome de angelman
CA3113648A1 (en) * 2018-09-21 2020-03-26 University Of Connecticut Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome
CA3133294A1 (en) * 2019-03-15 2020-09-24 Mir Scientific, Llc Methods for predicting prostate cancer and uses thereof

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KR20240155345A (ko) 2024-10-28
MX2024010820A (es) 2024-12-06
IL315359A (en) 2024-11-01
CN119384504A (zh) 2025-01-28
WO2023172535A2 (en) 2023-09-14
CA3259398A1 (en) 2023-09-14
AU2023231550A1 (en) 2024-09-26
EP4473116A2 (en) 2024-12-11
US20230332150A1 (en) 2023-10-19
WO2023172535A3 (en) 2023-11-30

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