JP7850136B2 - Wwox関連疾患の治療方法 - Google Patents

Wwox関連疾患の治療方法

Info

Publication number
JP7850136B2
JP7850136B2 JP2023509534A JP2023509534A JP7850136B2 JP 7850136 B2 JP7850136 B2 JP 7850136B2 JP 2023509534 A JP2023509534 A JP 2023509534A JP 2023509534 A JP2023509534 A JP 2023509534A JP 7850136 B2 JP7850136 B2 JP 7850136B2
Authority
JP
Japan
Prior art keywords
wwox
promoter
mice
brain
composition according
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.)
Active
Application number
JP2023509534A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023537093A5 (enExample
JP2023537093A (ja
Inventor
ラミ アケラン
スリーニヴァース レプディ
Original Assignee
イッサム リサーチ デベロップメント カンパニー オブ ザ ヘブライ ユニバーシティ オブ エルサレム リミテッド
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 イッサム リサーチ デベロップメント カンパニー オブ ザ ヘブライ ユニバーシティ オブ エルサレム リミテッド filed Critical イッサム リサーチ デベロップメント カンパニー オブ ザ ヘブライ ユニバーシティ オブ エルサレム リミテッド
Publication of JP2023537093A publication Critical patent/JP2023537093A/ja
Publication of JP2023537093A5 publication Critical patent/JP2023537093A5/ja
Application granted granted Critical
Publication of JP7850136B2 publication Critical patent/JP7850136B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0006Oxidoreductases (1.) acting on CH-OH groups as donors (1.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2023509534A 2020-08-11 2021-08-11 Wwox関連疾患の治療方法 Active JP7850136B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063064181P 2020-08-11 2020-08-11
US63/064,181 2020-08-11
US202163167277P 2021-03-29 2021-03-29
US63/167,277 2021-03-29
PCT/IL2021/050982 WO2022034591A1 (en) 2020-08-11 2021-08-11 Method for the treatment of wwox associated diseases

Publications (3)

Publication Number Publication Date
JP2023537093A JP2023537093A (ja) 2023-08-30
JP2023537093A5 JP2023537093A5 (enExample) 2024-08-20
JP7850136B2 true JP7850136B2 (ja) 2026-04-22

Family

ID=77910831

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023509534A Active JP7850136B2 (ja) 2020-08-11 2021-08-11 Wwox関連疾患の治療方法

Country Status (9)

Country Link
US (1) US20230293726A1 (enExample)
EP (2) EP4582143A3 (enExample)
JP (1) JP7850136B2 (enExample)
KR (1) KR20230051223A (enExample)
AU (1) AU2021324538A1 (enExample)
CA (1) CA3188895A1 (enExample)
ES (1) ES3032462T3 (enExample)
IL (1) IL300374A (enExample)
WO (1) WO2022034591A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116463413B (zh) * 2023-06-12 2023-10-03 佛山科学技术学院 猪间性病相关的wwox基因分子标记及其应用
WO2025264815A1 (en) * 2024-06-18 2025-12-26 Synaptec Network, Inc. Systems and methods of exosome mediated delivery of antibodies

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013510114A (ja) 2009-11-03 2013-03-21 ファーネクスト アルツハイマー病を処置するための新しい治療アプローチ
JP2014205696A (ja) 2008-04-29 2014-10-30 ファーネクストPharnext 細胞ストレス応答の調節を通したアルツハイマー病および関連障害の処置のための新たな治療アプローチ
WO2017147720A1 (en) 2016-03-04 2017-09-08 Exerkine Corporation Method for treating a central nervous system disorder
WO2020006125A1 (en) 2018-06-26 2020-01-02 Ribonova Inc. Methods of treating mitochondrial dysfunction
WO2021089856A1 (en) 2019-11-08 2021-05-14 Horama Modified adeno-associated virus vectors and delivery thereof into the central nervous system

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7060811B2 (en) * 2000-10-13 2006-06-13 Board Of Regents, The University Of Texas System WWOX: a tumor suppressor gene mutated in multiple cancers
US20090270484A1 (en) * 2005-10-05 2009-10-29 The Ohio State University Research Foundation WWOX Vectors and Uses in Treatment of Cancer
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014205696A (ja) 2008-04-29 2014-10-30 ファーネクストPharnext 細胞ストレス応答の調節を通したアルツハイマー病および関連障害の処置のための新たな治療アプローチ
JP2013510114A (ja) 2009-11-03 2013-03-21 ファーネクスト アルツハイマー病を処置するための新しい治療アプローチ
WO2017147720A1 (en) 2016-03-04 2017-09-08 Exerkine Corporation Method for treating a central nervous system disorder
WO2020006125A1 (en) 2018-06-26 2020-01-02 Ribonova Inc. Methods of treating mitochondrial dysfunction
WO2021089856A1 (en) 2019-11-08 2021-05-14 Horama Modified adeno-associated virus vectors and delivery thereof into the central nervous system

Also Published As

Publication number Publication date
ES3032462T3 (en) 2025-07-18
EP4582143A2 (en) 2025-07-09
KR20230051223A (ko) 2023-04-17
CA3188895A1 (en) 2022-02-17
EP4196595B1 (en) 2025-03-12
WO2022034591A1 (en) 2022-02-17
JP2023537093A (ja) 2023-08-30
EP4196595A1 (en) 2023-06-21
US20230293726A1 (en) 2023-09-21
IL300374A (en) 2023-04-01
EP4582143A3 (en) 2025-10-22
AU2021324538A1 (en) 2023-03-09

Similar Documents

Publication Publication Date Title
Moyon et al. TET1-mediated DNA hydroxymethylation regulates adult remyelination in mice
Steinberg et al. Modeling genetic epileptic encephalopathies using brain organoids
Mei et al. Stage-specific deletion of Olig2 conveys opposing functions on differentiation and maturation of oligodendrocytes
Repudi et al. Neuronal deletion of Wwox, associated with WOREE syndrome, causes epilepsy and myelin defects
Lang et al. Adenomatous polyposis coli regulates oligodendroglial development
Zhu et al. BAI1 regulates spatial learning and synaptic plasticity in the hippocampus
Tung et al. Mir-17∼ 92 confers motor neuron subtype differential resistance to ALS-associated degeneration
US20230287368A1 (en) Crispr-cas system for gene therapy
JP7850136B2 (ja) Wwox関連疾患の治療方法
US20210228741A1 (en) Methods and compositions to stimulate retinal regeneration
Pulcrano et al. miR-218 promotes dopaminergic differentiation and controls neuron excitability and neurotransmitter release through the regulation of a synaptic-related genes network
CN111686124B (zh) miR-486-3p在制备治疗SAH导致的神经炎症产品中的应用
WO2023069484A1 (en) Microrna-mediated methods for rejuvenating cns glial populations
Zhou et al. MiR-138 reprograms dental pulp stem cells into GABAergic neurons via the GATAD2B/MTA3/WNTs axis for stroke treatment
Mauriac et al. AAV-mediated exon skipping therapy for Usher syndrome, type 2A
CN116457025A (zh) 用于治疗wwox相关疾病的方法
Fu et al. Comparative transcriptomic profiling reveals a role for Olig1 in promoting axon regeneration
US20230270818A1 (en) Tcf7l2 mediated remyelination in the brain
Alarcon-Martineza et al. Characterization of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina
Abati In vivo and in vitro evaluation of the combination of RNA interfering and gene therapy for treating Mitofusin2-related diseases
Wang Neuronal Conversion of Human Glioblastoma Cells by Single Neuronal Transcription Factor
Baughn Therapeutic Restoration of Stathmin-2 RNA Processing in TDP-43 Proteinopathies
Obeid et al. Neuron-Specific WWOX Gene Therapy Produces Dose-Dependent, Durable Rescue in a Model of WWOX-Related Epileptic Encephalopathy
Croci CRISPR-Cas9 gene editing: a new promising treatment for Rett syndrome
JP2026505771A (ja) グリア細胞の競合的置換

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20230406

RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20230406

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240809

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240809

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251007

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251225

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20260324

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20260410

R150 Certificate of patent or registration of utility model

Ref document number: 7850136

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150