CN119255814A - 脑中bag3和蛋白质的质量控制 - Google Patents

脑中bag3和蛋白质的质量控制 Download PDF

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Publication number
CN119255814A
CN119255814A CN202380037961.7A CN202380037961A CN119255814A CN 119255814 A CN119255814 A CN 119255814A CN 202380037961 A CN202380037961 A CN 202380037961A CN 119255814 A CN119255814 A CN 119255814A
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CN
China
Prior art keywords
bag3
vector
brain
expression
promoter
Prior art date
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Pending
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CN202380037961.7A
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English (en)
Chinese (zh)
Inventor
亚瑟·M·费尔德曼
瓦莱丽·迈尔斯
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Federal Higher Education System Temple University
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Federal Higher Education System Temple University
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Publication of CN119255814A publication Critical patent/CN119255814A/zh
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—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
    • C07K14/46—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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Psychiatry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Hospice & Palliative Care (AREA)
  • Microbiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202380037961.7A 2022-05-03 2023-05-02 脑中bag3和蛋白质的质量控制 Pending CN119255814A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263337914P 2022-05-03 2022-05-03
US63/337,914 2022-05-03
PCT/US2023/020659 WO2023215266A1 (en) 2022-05-03 2023-05-02 Bag3 and protein quality control in the brain

Publications (1)

Publication Number Publication Date
CN119255814A true CN119255814A (zh) 2025-01-03

Family

ID=88646932

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380037961.7A Pending CN119255814A (zh) 2022-05-03 2023-05-02 脑中bag3和蛋白质的质量控制

Country Status (11)

Country Link
US (1) US20250289858A1 (enExample)
EP (1) EP4518882A4 (enExample)
JP (1) JP2025514489A (enExample)
KR (1) KR20250010008A (enExample)
CN (1) CN119255814A (enExample)
AU (1) AU2023265031A1 (enExample)
CA (1) CA3250880A1 (enExample)
CL (1) CL2024003318A1 (enExample)
IL (1) IL316559A (enExample)
MX (1) MX2024013495A (enExample)
WO (1) WO2023215266A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120478386A (zh) * 2025-07-18 2025-08-15 南京大学 一种用于预防、改善和/或治疗阿尔茨海默症的siRNA及其用途

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024265705A1 (en) * 2023-05-02 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1323733A1 (en) * 2001-12-28 2003-07-02 Arturo Leone BAG3 nucleotide and protein sequences to be used in research, diagnostics and therapy for cell death-involving diseases
WO2015117010A2 (en) * 2014-01-31 2015-08-06 Temple University Of The Commonwealth System Of Higher Education Bag3 as a target for therapy of heart failure
CN109937045A (zh) * 2016-08-11 2019-06-25 天普大学-联邦高等教育系统 局部缺血/再灌注损伤
US20210353613A1 (en) * 2018-10-19 2021-11-18 Aclipse One Inc. Treatment of neurological disease
JP7595006B2 (ja) * 2018-11-04 2024-12-05 フィジーン、エルエルシー 脳卒中、慢性外傷性脳症、及び外傷性脳損傷を含む脳性低酸素症の治療
US20220088127A1 (en) * 2019-01-15 2022-03-24 Temple University - Of The Commonwealth System Of Higher Education Targeting the bag family for the treatment of neurodegenerative disease
WO2023069744A1 (en) * 2021-10-22 2023-04-27 Temple University Of The Commonwealth System Of Higher Education Bag3 methods and uses for treatment of inflammation

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120478386A (zh) * 2025-07-18 2025-08-15 南京大学 一种用于预防、改善和/或治疗阿尔茨海默症的siRNA及其用途
CN120478386B (zh) * 2025-07-18 2025-10-17 南京大学 一种用于预防、改善和/或治疗阿尔茨海默症的siRNA及其用途

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Publication number Publication date
JP2025514489A (ja) 2025-05-02
EP4518882A1 (en) 2025-03-12
IL316559A (en) 2024-12-01
CA3250880A1 (en) 2023-11-09
US20250289858A1 (en) 2025-09-18
KR20250010008A (ko) 2025-01-20
EP4518882A4 (en) 2026-04-22
WO2023215266A1 (en) 2023-11-09
AU2023265031A1 (en) 2024-11-28
MX2024013495A (es) 2025-02-10
CL2024003318A1 (es) 2025-03-21

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