CN120225677A - 光响应改造型视蛋白类 - Google Patents

光响应改造型视蛋白类 Download PDF

Info

Publication number
CN120225677A
CN120225677A CN202380062525.5A CN202380062525A CN120225677A CN 120225677 A CN120225677 A CN 120225677A CN 202380062525 A CN202380062525 A CN 202380062525A CN 120225677 A CN120225677 A CN 120225677A
Authority
CN
China
Prior art keywords
amino acid
protein
acid sequence
opsin
sequence
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.)
Pending
Application number
CN202380062525.5A
Other languages
English (en)
Chinese (zh)
Inventor
山下高广
酒井佳寿美
七田芳则
佐藤惠太
青木诗音
坚田侑作
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ruishiwei Co
Kyoto University NUC
Original Assignee
Ruishiwei Co
Kyoto University NUC
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 Ruishiwei Co, Kyoto University NUC filed Critical Ruishiwei Co
Publication of CN120225677A publication Critical patent/CN120225677A/zh
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • 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
    • 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
    • A61K48/0075Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/72Receptors; Cell surface antigens; Cell surface determinants for hormones
    • C07K14/723G protein coupled receptor, e.g. TSHR-thyrotropin-receptor, LH/hCG receptor, FSH receptor
    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0618Cells of the nervous system
    • C12N5/0619Neurons
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2510/00Genetically modified cells
    • 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
    • C12N2529/00Culture process characterised by the use of electromagnetic stimulation
    • C12N2529/10Stimulation by light
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Cell Biology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Endocrinology (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202380062525.5A 2022-08-31 2023-08-30 光响应改造型视蛋白类 Pending CN120225677A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2022-138707 2022-08-31
JP2022138707 2022-08-31
PCT/JP2023/031644 WO2024048688A1 (ja) 2022-08-31 2023-08-30 光応答改変型オプシン類

Publications (1)

Publication Number Publication Date
CN120225677A true CN120225677A (zh) 2025-06-27

Family

ID=90099743

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380062525.5A Pending CN120225677A (zh) 2022-08-31 2023-08-30 光响应改造型视蛋白类

Country Status (5)

Country Link
EP (1) EP4582544A1 (https=)
JP (1) JPWO2024048688A1 (https=)
KR (1) KR20250061742A (https=)
CN (1) CN120225677A (https=)
WO (1) WO2024048688A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025214408A1 (en) * 2024-04-10 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Optogenetic modulation for vision restoration

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585108A (en) 1994-12-30 1996-12-17 Nanosystems L.L.C. Formulations of oral gastrointestinal therapeutic agents in combination with pharmaceutically acceptable clays
CA2224907A1 (en) 1995-07-25 1997-02-13 Introgene B.V. Methods and means for targeted gene delivery
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
JP3521382B2 (ja) 1997-02-27 2004-04-19 日本たばこ産業株式会社 細胞間接着及びシグナル伝達を媒介する細胞表面分子
US6649138B2 (en) 2000-10-13 2003-11-18 Quantum Dot Corporation Surface-modified semiconductive and metallic nanoparticles having enhanced dispersibility in aqueous media
EP1474109B1 (en) 2001-12-21 2010-08-25 Alcon, Inc. Use of synthetic inorganic nanoparticles as carriers for ophthalmic drugs
CN102016814B (zh) 2005-06-17 2013-10-23 北卡罗来纳大学查珀尔希尔分校 纳米粒子制备方法、系统及材料
US9782342B2 (en) 2010-10-11 2017-10-10 Wichita State University Composite magnetic nanoparticle drug delivery system
WO2012125987A2 (en) 2011-03-17 2012-09-20 Massachusetts Institute Of Technology Delivery system
US20120283503A1 (en) 2011-04-29 2012-11-08 The Johns Hopkins University Nanoparticle loaded stem cells and their use in mri guided hyperthermia
WO2020206098A1 (en) * 2019-04-03 2020-10-08 Regenxbio Inc. Gene therapy for eye pathologies
JP7696675B2 (ja) 2021-03-10 2025-06-23 ダイハツ工業株式会社 車両用制御装置

Also Published As

Publication number Publication date
EP4582544A1 (en) 2025-07-09
WO2024048688A1 (ja) 2024-03-07
JPWO2024048688A1 (https=) 2024-03-07
KR20250061742A (ko) 2025-05-08

Similar Documents

Publication Publication Date Title
Ganjawala et al. Improved CoChR variants restore visual acuity and contrast sensitivity in a mouse model of blindness under ambient light conditions
EP2465931B1 (en) Light-receiving channel rhodopsin having improved expression efficiency
US12606606B2 (en) Nucleic acid construct that encodes chimeric rhodopsin
JP7299632B2 (ja) 網膜疾患の予防および進行抑制、視覚認知行動機能の改善、および視覚機能強化
US20200030458A1 (en) Gene Therapy for the Treatment of CNGB1-linked Retinitis Pigmentosa
EP3508212B1 (en) Rhodopsin gene therapy for regenerating visual function or for preventing deterioration in visual function
Pang et al. Achromatopsia as a potential candidate for gene therapy
Fu et al. In vivo prime editing rescues photoreceptor degeneration in nonsense mutant retinitis pigmentosa
Nojima et al. A point mutation in Semaphorin 4A associates with defective endosomal sorting and causes retinal degeneration
CN120225677A (zh) 光响应改造型视蛋白类
US11771741B2 (en) Nucleic acid construct that encodes chimeric rhodopsin
Narfström et al. The domestic cat as a large animal model for characterization of disease and therapeutic intervention in hereditary retinal blindness
WO2022059736A1 (ja) 小胞体ストレスまたはオールトランスレチナールに関連する疾患、障害または症状を治療または予防するため、または網膜厚を保護し、または網膜厚の萎縮もしくは萎縮進行を抑制するための組成物。
CN120309704A (zh) 新型光敏感通道蛋白vr2.0及其用途
HK40124146A (zh) 光响应改造型视蛋白类
JPWO2020054868A1 (ja) 光応答性タンパク質及びその利用
CN115397991B (zh) 用于颜色识别的光响应性蛋白及其应用
HK40097616A (en) Composition for treating or preventing diseases, disorders, or conditions associated with endoplasmic reticulum stress or all-trans-retinal, protecting retinal thickness, or suppressing reduction in retinal thickness or progress of reduction in retinal thickness
HK40097920A (zh) 用於治疗或预防与内质网应激或全反式视黄醛相关的疾病、障碍或症状、或者用於保护视网膜厚度、或者用於抑制视网膜厚度的萎缩或萎缩进展的组合物
CN115335525B (zh) 经修饰的通道视紫红质
CN120309705A (zh) 新型光敏感通道蛋白vr3.0及其用途
Cehajic-Kapetanovic Restoring Vision Using Human Opsins

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40124146

Country of ref document: HK