IL288462A - AAV vectors with a promoter for zero myelin protein and their use for the treatment of diseases associated with Schwann cells such as Charcot-Marie-Toth disease - Google Patents

AAV vectors with a promoter for zero myelin protein and their use for the treatment of diseases associated with Schwann cells such as Charcot-Marie-Toth disease

Info

Publication number
IL288462A
IL288462A IL288462A IL28846221A IL288462A IL 288462 A IL288462 A IL 288462A IL 288462 A IL288462 A IL 288462A IL 28846221 A IL28846221 A IL 28846221A IL 288462 A IL288462 A IL 288462A
Authority
IL
Israel
Prior art keywords
charcot
marie
aav vectors
associated diseases
schwann cell
Prior art date
Application number
IL288462A
Other languages
English (en)
Hebrew (he)
Original Assignee
The Cyprus Foundation For Muscular Dystrophy Res
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 The Cyprus Foundation For Muscular Dystrophy Res filed Critical The Cyprus Foundation For Muscular Dystrophy Res
Publication of IL288462A publication Critical patent/IL288462A/en

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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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • 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
    • 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
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A01K2227/00—Animals characterised by species
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
IL288462A 2019-06-03 2021-11-28 AAV vectors with a promoter for zero myelin protein and their use for the treatment of diseases associated with Schwann cells such as Charcot-Marie-Toth disease IL288462A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1907882.3A GB201907882D0 (en) 2019-06-03 2019-06-03 Methods
PCT/EP2020/065312 WO2020245169A1 (en) 2019-06-03 2020-06-03 Aav vectors with myelin protein zero promoter and uses thereof for treating schwann cell-associated diseases like charcot-marie-tooth disease

Publications (1)

Publication Number Publication Date
IL288462A true IL288462A (en) 2022-01-01

Family

ID=67385825

Family Applications (1)

Application Number Title Priority Date Filing Date
IL288462A IL288462A (en) 2019-06-03 2021-11-28 AAV vectors with a promoter for zero myelin protein and their use for the treatment of diseases associated with Schwann cells such as Charcot-Marie-Toth disease

Country Status (16)

Country Link
US (1) US12582725B2 (https=)
EP (1) EP3976800A1 (https=)
JP (1) JP7692369B2 (https=)
KR (1) KR20220016485A (https=)
CN (1) CN114026242A (https=)
AU (1) AU2020288605A1 (https=)
BR (1) BR112021024285A2 (https=)
CA (1) CA3142211A1 (https=)
CL (1) CL2021003201A1 (https=)
CO (1) CO2021016487A2 (https=)
EA (1) EA202290001A1 (https=)
GB (1) GB201907882D0 (https=)
IL (1) IL288462A (https=)
MX (1) MX2021014789A (https=)
SG (1) SG11202112889VA (https=)
WO (1) WO2020245169A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240076665A1 (en) * 2020-12-22 2024-03-07 Wisconsin Alumni Research Foundation Regulatory elements for schwann cell-specific gene expression
CA3234702A1 (en) 2021-10-07 2023-04-13 Research Institute At Nationwide Children's Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
JP2025502269A (ja) * 2022-01-13 2025-01-24 ツールゲン インコーポレイテッド シュワン細胞特異的プロモーター
GB202303495D0 (en) * 2023-03-09 2023-04-26 Ucl Business Ltd Therapy for charcot-marie-tooth disease
WO2025105734A1 (ko) * 2023-11-15 2025-05-22 주식회사 카인사이언스 탈수초 질환 치료용 펩타이드 및 이의 용도
WO2025174135A1 (ko) * 2024-02-16 2025-08-21 주식회사 엣진 샤르코-마리-투스 질환 치료를 위한 징크 핑거 단백질 및 이를 발현시키는 방법
CN118291543A (zh) * 2024-02-29 2024-07-05 神拓生物技术(杭州)有限公司 一种二型神经纤维瘤基因治疗的慢病毒载体及其应用
KR20260001543A (ko) * 2024-06-25 2026-01-06 연세대학교 산학협력단 바이러스 벡터를 포함한 난청 치료용 조성물

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PL210751B1 (pl) 2006-09-12 2012-02-29 Anna Jurewicz Izolowany fragment białka Nogo-A, jego zastosowanie oraz sposób diagnozowania oraz leczenia stwardnienia rozsianego i polineuropatii zapalno-demielinizacyjnej
US20090018031A1 (en) 2006-12-07 2009-01-15 Switchgear Genomics Transcriptional regulatory elements of biological pathways tools, and methods
US20090215178A1 (en) 2008-02-22 2009-08-27 Zequn Tang Methods to enhance the stability and homogeneity of transgene expression in clonal cell lines
CA2730785A1 (en) 2008-07-16 2010-01-21 The Board Of Trustees Of The Leland Stanford Junior University Modulation of gm98 (mrf) in remyelination
AU2010289419A1 (en) 2009-09-02 2012-03-29 The University Of Chicago Methods and systems for inducible ablation of neural cells
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
AU2012258558A1 (en) 2011-05-26 2013-05-02 Biogen Ma Inc. Methods of treating multiple sclerosis and preserving and/or increasing myelin content
DK2820042T3 (da) 2012-02-29 2019-10-28 Baxalta GmbH Igg-stimuleret remyelinisering af perifere nerver
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IL299762A (en) 2020-07-13 2023-03-01 Univ Pennsylvania Useful preparations for the treatment of Charcot-Marietot disease

Also Published As

Publication number Publication date
KR20220016485A (ko) 2022-02-09
BR112021024285A2 (pt) 2022-01-11
CN114026242A (zh) 2022-02-08
JP7692369B2 (ja) 2025-06-13
SG11202112889VA (en) 2021-12-30
CO2021016487A2 (es) 2022-01-17
CA3142211A1 (en) 2020-12-10
EP3976800A1 (en) 2022-04-06
MX2021014789A (es) 2022-02-11
WO2020245169A1 (en) 2020-12-10
US20220323611A1 (en) 2022-10-13
AU2020288605A1 (en) 2022-02-03
CL2021003201A1 (es) 2023-02-03
EA202290001A1 (ru) 2022-03-28
US12582725B2 (en) 2026-03-24
JP2022535412A (ja) 2022-08-08
GB201907882D0 (en) 2019-07-17

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