CN107427666B - 脊髓软膜下基因递送系统 - Google Patents

脊髓软膜下基因递送系统 Download PDF

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CN107427666B
CN107427666B CN201580078566.9A CN201580078566A CN107427666B CN 107427666 B CN107427666 B CN 107427666B CN 201580078566 A CN201580078566 A CN 201580078566A CN 107427666 B CN107427666 B CN 107427666B
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subpial
delivery system
aav9
gene delivery
spinal cord
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CN107427666A (zh
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马丁.马尔萨拉
宮之原厚司
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University of California San Diego UCSD
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • 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
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61L—METHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L29/00—Materials for catheters, medical tubing, cannulae, or endoscopes or for coating catheters
    • A61L29/04—Macromolecular materials
    • A61L29/041—Macromolecular materials obtained by reactions only involving carbon-to-carbon unsaturated bonds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61M25/00—Catheters; Hollow probes
    • A61M25/01—Introducing, guiding, advancing, emplacing or holding catheters
    • A61M25/06—Body-piercing guide needles or the like
    • A61M25/0662—Guide tubes
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
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    • A61P25/20—Hypnotics; Sedatives
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    • 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
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    • A61B—DIAGNOSIS; SURGERY; IDENTIFICATION
    • A61B17/00—Surgical instruments, devices or methods
    • A61B17/34—Trocars; Puncturing needles
    • A61B17/3401—Puncturing needles for the peridural or subarachnoid space or the plexus, e.g. for anaesthesia
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    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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CN201580078566.9A 2015-01-30 2015-12-15 脊髓软膜下基因递送系统 Active CN107427666B (zh)

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US201562110340P 2015-01-30 2015-01-30
US62/110,340 2015-01-30
PCT/US2015/065704 WO2016122791A1 (en) 2015-01-30 2015-12-15 Spinal subpial gene delivery system

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CN107427666B true CN107427666B (zh) 2022-11-04

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EP (1) EP3250279B1 (https=)
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CA2975447A1 (en) 2016-08-04
JP2025015673A (ja) 2025-01-30
EP3250279A4 (en) 2018-06-20
JP2020019796A (ja) 2020-02-06
JP2021181456A (ja) 2021-11-25
HK1247867A1 (zh) 2018-10-05
EP3250279B1 (en) 2020-08-19
CN107427666A (zh) 2017-12-01
WO2016122791A1 (en) 2016-08-04
CA2975447C (en) 2021-02-23
EP3250279A1 (en) 2017-12-06
JP2018506530A (ja) 2018-03-08
US20180008727A1 (en) 2018-01-11

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