CN107530449A - 改善视觉的基因疗法 - Google Patents

改善视觉的基因疗法 Download PDF

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Publication number
CN107530449A
CN107530449A CN201680023264.6A CN201680023264A CN107530449A CN 107530449 A CN107530449 A CN 107530449A CN 201680023264 A CN201680023264 A CN 201680023264A CN 107530449 A CN107530449 A CN 107530449A
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carrier
rod
used according
aav
light
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CN201680023264.6A
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English (en)
Chinese (zh)
Inventor
M·瑞兹
R·阿里
A·史密斯
K·西口
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UCL Business Ltd
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UCL Business Ltd
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    • 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
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201680023264.6A 2015-02-23 2016-02-19 改善视觉的基因疗法 Pending CN107530449A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1503008.3 2015-02-23
GB201503008A GB201503008D0 (en) 2015-02-23 2015-02-23 Treatment
PCT/GB2016/050419 WO2016135457A1 (en) 2015-02-23 2016-02-19 Gene therapy to improve vision

Publications (1)

Publication Number Publication Date
CN107530449A true CN107530449A (zh) 2018-01-02

Family

ID=52822045

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201680023264.6A Pending CN107530449A (zh) 2015-02-23 2016-02-19 改善视觉的基因疗法

Country Status (29)

Country Link
US (3) US20180030477A1 (enExample)
EP (1) EP3261679B1 (enExample)
JP (1) JP6771474B2 (enExample)
KR (1) KR102272466B1 (enExample)
CN (1) CN107530449A (enExample)
AU (2) AU2016225277B2 (enExample)
CA (1) CA2977464C (enExample)
CY (1) CY1126098T1 (enExample)
DK (1) DK3261679T3 (enExample)
EA (1) EA201791900A1 (enExample)
ES (1) ES2947308T3 (enExample)
FI (1) FI3261679T3 (enExample)
GB (1) GB201503008D0 (enExample)
HK (1) HK1248594A1 (enExample)
HR (1) HRP20230775T1 (enExample)
HU (1) HUE062319T2 (enExample)
IL (1) IL254066B (enExample)
LT (1) LT3261679T (enExample)
MX (1) MX2017010772A (enExample)
MY (1) MY186444A (enExample)
PH (1) PH12017501525B1 (enExample)
PL (1) PL3261679T3 (enExample)
PT (1) PT3261679T (enExample)
RS (1) RS64372B1 (enExample)
SG (2) SG11201706822XA (enExample)
SI (1) SI3261679T1 (enExample)
SM (1) SMT202300200T1 (enExample)
WO (1) WO2016135457A1 (enExample)
ZA (1) ZA201706440B (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3037436A1 (en) * 2016-09-22 2018-03-29 Sorbonne Universite Optogenetically transformed photoreceptor precursor cells for the use in the treatment of retinal degenerative diseases
WO2023280388A1 (en) * 2021-07-06 2023-01-12 Institute Of Molecular And Clinical Ophthalmology Basel Promoter for specific expression of genes in rod photoreceptors

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101484005A (zh) * 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
CN101970013A (zh) * 2008-04-18 2011-02-09 诺瓦提斯研究基金会弗里德里克·米谢尔生物医学研究所 治疗失明的新型治疗工具和方法

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2442670A1 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
GB2498325B (en) * 2010-10-15 2018-05-02 Eos Neuroscience Inc Engineered human GRM6 enhancer sequence for use in eye therapies
US8957028B2 (en) * 2010-11-13 2015-02-17 Massachusetts Institute Of Technology Red-shifted opsin molecules and uses thereof
WO2012167109A2 (en) * 2011-06-03 2012-12-06 Massachusetts Eye & Ear Infirmary Rpgrip1 gene therapy for leber congenital amaurosis
WO2013124477A1 (en) * 2012-02-24 2013-08-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions for treatment of retinal degenerative diseases
AU2015296502A1 (en) * 2014-07-29 2017-02-23 Circuit Therapeutics, Inc. System and method for optogenetic therapy

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101484005A (zh) * 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
CN101970013A (zh) * 2008-04-18 2011-02-09 诺瓦提斯研究基金会弗里德里克·米谢尔生物医学研究所 治疗失明的新型治疗工具和方法

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
I SANDOVAL ET AL: "AVV-directed overexpression of R9AP and RGS9-1 in Rat Retina", 《INVESTIGATIVE OPHTHALMOLOGY&VISUAL SCIENCE》 *
M.E.BURNS ET AL: "Lessons from photoreceptors:turning off G-protein signaling in living cells", 《PHYSIOLOGY》 *
XUE HAN ET AL: "A high-light sensitivity optical neural silencer:development and application of optogenetic control of non-human primate cortex", 《FRONTIERS IN SYSTEMS NEUROSCIENCE》 *
蒋沁等: "视网膜色素变性的研究进展", 《眼视光学杂质》 *

Also Published As

Publication number Publication date
JP2018506982A (ja) 2018-03-15
AU2016225277B2 (en) 2021-12-09
SMT202300200T1 (it) 2023-09-06
CY1126098T1 (el) 2023-11-15
US20250290096A1 (en) 2025-09-18
GB201503008D0 (en) 2015-04-08
SI3261679T1 (sl) 2023-09-29
MX2017010772A (es) 2018-03-01
CA2977464C (en) 2022-04-05
EA201791900A1 (ru) 2018-02-28
WO2016135457A1 (en) 2016-09-01
HUE062319T2 (hu) 2023-10-28
FI3261679T3 (fi) 2023-06-28
ZA201706440B (en) 2021-06-30
US20200377907A1 (en) 2020-12-03
BR112017018062A2 (pt) 2018-04-10
PH12017501525B1 (en) 2023-08-30
SG10202005923WA (en) 2020-07-29
KR102272466B1 (ko) 2021-07-02
IL254066A0 (en) 2017-10-31
AU2022201553A1 (en) 2022-03-31
HK1248594A1 (zh) 2018-10-19
AU2016225277A1 (en) 2017-10-12
RS64372B1 (sr) 2023-08-31
HRP20230775T1 (hr) 2023-10-27
US20180030477A1 (en) 2018-02-01
SG11201706822XA (en) 2017-09-28
LT3261679T (lt) 2023-07-10
JP6771474B2 (ja) 2020-10-21
IL254066B (en) 2021-10-31
PL3261679T3 (pl) 2023-09-04
DK3261679T3 (da) 2023-06-26
HK1248595A1 (en) 2018-10-19
EP3261679B1 (en) 2023-05-03
AU2022201553B2 (en) 2024-08-15
PT3261679T (pt) 2023-07-04
MY186444A (en) 2021-07-22
PH12017501525A1 (en) 2018-02-05
CA2977464A1 (en) 2016-09-01
ES2947308T3 (es) 2023-08-04
EP3261679A1 (en) 2018-01-03
NZ735735A (en) 2021-09-24
KR20180012737A (ko) 2018-02-06

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