JP2023553005A - 末梢ミエリンタンパク質２２の発現の阻害のための生成物及び方法 - Google Patents

末梢ミエリンタンパク質２２の発現の阻害のための生成物及び方法 Download PDF

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JP2023553005A
JP2023553005A JP2023534050A JP2023534050A JP2023553005A JP 2023553005 A JP2023553005 A JP 2023553005A JP 2023534050 A JP2023534050 A JP 2023534050A JP 2023534050 A JP2023534050 A JP 2023534050A JP 2023553005 A JP2023553005 A JP 2023553005A
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JP2023553005A5 (https=
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クエントン ハーパー、スコット
クレオパ、クレオパス
スタヴロウ、マリナ
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Cyprus Foundation For Muscular Dystrophy Research D/b/a Cyprus Institute Of Neurology And Genetics
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Cyprus Foundation For Muscular Dystrophy Research D/b/a Cyprus Institute Of Neurology And Genetics
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Publication of JP2023553005A5 publication Critical patent/JP2023553005A5/ja
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Virology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2023534050A 2020-12-01 2021-11-30 末梢ミエリンタンパク質２２の発現の阻害のための生成物及び方法 Pending JP2023553005A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063120190P 2020-12-01 2020-12-01
US63/120,190 2020-12-01
PCT/US2021/061177 WO2022119826A1 (en) 2020-12-01 2021-11-30 Products and methods for inhibition of expression of peripheral myelin protein-22

Publications (2)

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JP2023553005A true JP2023553005A (ja) 2023-12-20
JP2023553005A5 JP2023553005A5 (https=) 2024-11-18

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JP2023534050A Pending JP2023553005A (ja) 2020-12-01 2021-11-30 末梢ミエリンタンパク質２２の発現の阻害のための生成物及び方法

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Country Link
US (1) US20240318172A1 (https=)
EP (1) EP4256069A4 (https=)
JP (1) JP2023553005A (https=)
KR (1) KR20230134477A (https=)
CN (1) CN116917492A (https=)
AU (1) AU2021390471A1 (https=)
CA (1) CA3203748A1 (https=)
IL (1) IL303308A (https=)
WO (1) WO2022119826A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240103029A (ko) * 2021-11-18 2024-07-03 노파르티스 아게 샤르코 마리 투스 질환 치료용 pmp22 표적화 화합물
WO2024229211A2 (en) 2023-05-02 2024-11-07 Research Institute At Nationwide Children's Hospital A modular system to convert therapeutic microrna expression cassettes from polymerase iii-based to polymerase ii-based promoters
WO2025150052A1 (en) * 2024-01-12 2025-07-17 Potentia Ltd. Targeted delivery of drug conjugates to schwann cells and treatment methods in schwann cell-related diseases
WO2025242147A1 (zh) * 2024-05-21 2025-11-27 北京安龙生物医药有限公司 靶向外周髓鞘蛋白-22基因的寡核苷酸及其用途

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180066257A1 (en) * 2016-09-05 2018-03-08 Samsung Life Public Welfare Foundation Pharmaceutical composition for treating charcot marie tooth disease
WO2019066490A2 (ko) * 2017-09-28 2019-04-04 주식회사 툴젠 유전자 발현 조절을 위한 인위적인 게놈 조작
WO2020047268A1 (en) * 2018-08-29 2020-03-05 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of mutant gars protein
WO2020064749A1 (en) * 2018-09-25 2020-04-02 Centre National De La Recherche Scientifique Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease
KR20200124038A (ko) * 2019-04-23 2020-11-02 사회복지법인 삼성생명공익재단 마이크로rna-381을 유효성분으로 포함하는, pmp22 과발현에 의한 말초신경병증의 예방 또는 치료용 약학적 조성물

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012145597A2 (en) * 2011-04-21 2012-10-26 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
WO2017156242A1 (en) * 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression
JP7305534B2 (ja) * 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
AU2018224380A1 (en) * 2017-02-22 2019-08-29 Crispr Therapeutics Ag Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (PCSK9)-related disorders
JP7561129B2 (ja) * 2018-12-21 2024-10-03 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｐｍｐ２２の発現を低減するための化合物及び方法

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180066257A1 (en) * 2016-09-05 2018-03-08 Samsung Life Public Welfare Foundation Pharmaceutical composition for treating charcot marie tooth disease
WO2019066490A2 (ko) * 2017-09-28 2019-04-04 주식회사 툴젠 유전자 발현 조절을 위한 인위적인 게놈 조작
WO2020047268A1 (en) * 2018-08-29 2020-03-05 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of mutant gars protein
WO2020064749A1 (en) * 2018-09-25 2020-04-02 Centre National De La Recherche Scientifique Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease
KR20200124038A (ko) * 2019-04-23 2020-11-02 사회복지법인 삼성생명공익재단 마이크로rna-381을 유효성분으로 포함하는, pmp22 과발현에 의한 말초신경병증의 예방 또는 치료용 약학적 조성물

Also Published As

Publication number Publication date
KR20230134477A (ko) 2023-09-21
IL303308A (en) 2023-07-01
AU2021390471A9 (en) 2023-08-17
EP4256069A1 (en) 2023-10-11
AU2021390471A1 (en) 2023-06-29
CA3203748A1 (en) 2022-06-09
CN116917492A (zh) 2023-10-20
US20240318172A1 (en) 2024-09-26
EP4256069A4 (en) 2025-08-06
WO2022119826A1 (en) 2022-06-09

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