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

Links

Classifications

    • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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
WO2022140521A1 (en) * 2020-12-22 2022-06-30 Wisconsin Alumni Research Foundation Regulatory elements for schwann cell-specific gene expression
EP4413131A1 (en) 2021-10-07 2024-08-14 Research Institute at Nationwide Children's Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
WO2023136624A1 (ko) 2022-01-13 2023-07-20 주식회사 툴젠 슈반 세포 특이적 프로모터
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
KR20250126990A (ko) * 2024-02-16 2025-08-26 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

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2008073303A2 (en) 2006-12-07 2008-06-19 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
AU2009271524A1 (en) 2008-07-16 2010-01-21 The Board Of Trustees Of The Leland Stanford Junior University Modulation of GM98 (MRF) in remyelination
US20120233713A1 (en) 2009-09-02 2012-09-13 Brian Popko Methods and systems for inducible ablation of neural cells
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
EP2713724A4 (en) 2011-05-26 2015-03-11 Biogen Idec Inc METHODS OF TREATING MULTIPLE SCLEROSIS AND PRESERVING AND / OR INCREASING MYELINE CONTENT
CN107551269A (zh) 2012-02-29 2018-01-09 百深公司 周围神经的IgG刺激髓鞘再生
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9644215B2 (en) 2013-04-12 2017-05-09 The General Hospital Corporation AAV1-caspase gene therapy induced pyroptosis for the treatment of tumors
WO2015140351A1 (en) 2014-03-21 2015-09-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for enhancing myelination
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
CN114657141B (zh) 2014-09-12 2025-02-25 京都府公立大学法人 雪旺氏细胞及其制备方法
US20180305689A1 (en) 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use
JP6981973B2 (ja) 2015-10-01 2021-12-17 ヒート バイオロジクス，インコーポレイテッド Ｉ型及びｉｉ型細胞外ドメインを異種キメラタンパク質として連結する組成物及び方法
WO2018056412A1 (ja) 2016-09-23 2018-03-29 国立大学法人大阪大学 シュワン細胞分化促進剤及び末梢神経再生促進剤
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
KR102437228B1 (ko) 2016-11-14 2022-08-30 주식회사 툴젠 인위적으로 조작된 sc 기능 조절 시스템
WO2018106753A1 (en) 2016-12-07 2018-06-14 The General Hospital Corporation Methods and compositions relating the treatment of tumors
CN118581154A (zh) 2017-06-02 2024-09-03 国家健康与医学研究院 用于遗传障碍的基因疗法的基因组编辑手段和结合病毒载体的基因疗法
AU2018345772B2 (en) 2017-10-02 2025-04-17 Research Institute At Nationwide Children's Hospital MiRNA detargeting system for tissue specific interference
US11926653B2 (en) 2017-10-20 2024-03-12 Research Institute At Nationwide Children's Hospital Methods and materials for NT-3 gene therapy
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
BR112021024285A2 (pt) 2022-01-11
CN114026242A (zh) 2022-02-08
CL2021003201A1 (es) 2023-02-03
US12582725B2 (en) 2026-03-24
AU2020288605A1 (en) 2022-02-03
JP2022535412A (ja) 2022-08-08
JP7692369B2 (ja) 2025-06-13
CA3142211A1 (en) 2020-12-10
GB201907882D0 (en) 2019-07-17
EP3976800A1 (en) 2022-04-06
EA202290001A1 (ru) 2022-03-28
US20220323611A1 (en) 2022-10-13
WO2020245169A1 (en) 2020-12-10
MX2021014789A (es) 2022-02-11
SG11202112889VA (en) 2021-12-30
CO2021016487A2 (es) 2022-01-17
KR20220016485A (ko) 2022-02-09

Similar Documents

Publication Publication Date Title
IL288462A (en) 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
PT3740222T (pt) Uma proteína do capsid de raav modificada para terapia gênica
IL284554A (en) Structures for gene therapy to treat Wilson's disease
IL289518A (en) Recombinant ad35 vectors and improvements in gene therapy
IL277667A (en) Virus vectors for ocular tissues
IL283781A (en) Analosomes for the delivery of protein replacement therapeutics
IL289826A (en) Modified aav capsid proteins for the treatment of arthritis
EP4051324A4 (en) Gene therapy vectors
ZA202100859B (en) Recombinant protein variants
EP4041295A4 (en) MULTI-DOMAIN PROTEIN VACCINE
IL289667A (en) Cardiac aav gene therapy for cardiomyopathy in humans
EP4034550A4 (en) VACCINE THERAPY FOR RAN PROTEIN DISEASES
IL275951A (en) Vectors that evade the immune system and their use for gene therapy
IL290520A (en) Medicinal peptides
SG11202104178TA (en) Peptides and pharmaceutical compositions for treating eye diseases
IL285144A (en) Medicinal peptides
IL292247A (en) Methods of treating patients who have cfh mutations with recombinant cfh proteins
HK40061394A (en) Aav vectors with myelin protein zero promoter and uses thereof for treating schwann cell-associated diseases like charcot-marie-tooth disease
HUE065161T2 (hu) All-in-one AAV vektorok a coronavírus okozta betegségek kezelésére
HK40083253A (en) Adeno associated virus vectors for the treatment of hunter disease
HK40040300A (en) Therapeutic peptides and methods for treating autoimmune related disease
HK40074800A (en) Peptides and methods for treating diseases
GB201904810D0 (en) The treatment of protein aggregation diseases
HK40040931A (en) Immuno-evasive vectors and use for gene therapy
HK40077855A (en) Multi-domain protein vaccine