KR102625784B1 - 선조체 및 피질로의 바이러스 입자의 향상된 전달 - Google Patents

선조체 및 피질로의 바이러스 입자의 향상된 전달 Download PDF

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KR102625784B1
KR102625784B1 KR1020177025285A KR20177025285A KR102625784B1 KR 102625784 B1 KR102625784 B1 KR 102625784B1 KR 1020177025285 A KR1020177025285 A KR 1020177025285A KR 20177025285 A KR20177025285 A KR 20177025285A KR 102625784 B1 KR102625784 B1 KR 102625784B1
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raav
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aav
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KR20170110718A (ko
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리사 엠. 스타넥
라미아 시하버딘
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젠자임 코포레이션
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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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
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    • 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
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • 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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    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Psychiatry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Pain & Pain Management (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)
KR1020177025285A 2015-02-10 2016-02-09 선조체 및 피질로의 바이러스 입자의 향상된 전달 Active KR102625784B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020247000976A KR102863734B1 (ko) 2015-02-10 2016-02-09 선조체 및 피질로의 바이러스 입자의 향상된 전달

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562114544P 2015-02-10 2015-02-10
US62/114,544 2015-02-10
US201562220997P 2015-09-19 2015-09-19
US62/220,997 2015-09-19
PCT/US2016/017210 WO2016130591A2 (en) 2015-02-10 2016-02-09 Enhanced delivery of viral particles to the striatum and cortex

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KR1020247000976A Division KR102863734B1 (ko) 2015-02-10 2016-02-09 선조체 및 피질로의 바이러스 입자의 향상된 전달

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KR20170110718A KR20170110718A (ko) 2017-10-11
KR102625784B1 true KR102625784B1 (ko) 2024-01-15

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KR1020247000976A Active KR102863734B1 (ko) 2015-02-10 2016-02-09 선조체 및 피질로의 바이러스 입자의 향상된 전달

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US (3) US20190111157A1 (https=)
EP (3) EP4023758A1 (https=)
JP (4) JP6928558B2 (https=)
KR (2) KR102625784B1 (https=)
CN (2) CN116019934A (https=)
AU (3) AU2016219398A1 (https=)
BR (1) BR112017017037A2 (https=)
CA (1) CA2976082A1 (https=)
CL (1) CL2017002028A1 (https=)
CR (1) CR20170407A (https=)
DK (1) DK3256594T3 (https=)
EA (1) EA201791803A1 (https=)
ES (1) ES2904504T3 (https=)
HU (1) HUE057272T2 (https=)
IL (4) IL313840A (https=)
LT (1) LT3256594T (https=)
MX (2) MX388264B (https=)
NZ (1) NZ772772A (https=)
PH (1) PH12017501436A1 (https=)
PL (1) PL3256594T3 (https=)
PT (1) PT3256594T (https=)
RS (1) RS62811B1 (https=)
SG (3) SG10201907399RA (https=)
SI (1) SI3256594T1 (https=)
TN (1) TN2017000353A1 (https=)
TW (1) TWI730952B (https=)
WO (1) WO2016130591A2 (https=)

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