CN105770909A - 脊髓失调症的基因治疗 - Google Patents
脊髓失调症的基因治疗 Download PDFInfo
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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- 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
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- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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- 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
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- 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
- C07K14/475—Growth factors; Growth regulators
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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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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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
- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
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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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
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- Epidemiology (AREA)
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- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
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- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
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- Physics & Mathematics (AREA)
- Physical Education & Sports Medicine (AREA)
- Psychiatry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (4)
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| US67721305P | 2005-05-02 | 2005-05-02 | |
| US60/677,213 | 2005-05-02 | ||
| US79021706P | 2006-04-08 | 2006-04-08 | |
| US60/790,217 | 2006-04-08 |
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| CNA200680023365XA Division CN101208108A (zh) | 2005-05-02 | 2006-05-02 | 脊髓失调症的基因治疗 |
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| CN105770909A true CN105770909A (zh) | 2016-07-20 |
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| CN201610153759.2A Pending CN105770909A (zh) | 2005-05-02 | 2006-05-02 | 脊髓失调症的基因治疗 |
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| EP (1) | EP1879623B1 (https=) |
| JP (5) | JP5766389B2 (https=) |
| CN (1) | CN105770909A (https=) |
| AU (1) | AU2006242129A1 (https=) |
| BR (1) | BRPI0611540A2 (https=) |
| CA (1) | CA2606490C (https=) |
| DK (1) | DK1879623T3 (https=) |
| ES (1) | ES2394482T3 (https=) |
| IL (2) | IL187023A (https=) |
| MX (1) | MX2007013734A (https=) |
| PL (1) | PL1879623T3 (https=) |
| PT (1) | PT1879623E (https=) |
| WO (1) | WO2006119341A2 (https=) |
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| EP1879624B1 (en) | 2005-05-02 | 2011-09-21 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| EP1919497B1 (en) | 2005-07-22 | 2020-02-12 | The Board of Trustees of the Leland Stanford Junior University | Light-activated cation channel and uses thereof |
| US9238150B2 (en) * | 2005-07-22 | 2016-01-19 | The Board Of Trustees Of The Leland Stanford Junior University | Optical tissue interface method and apparatus for stimulating cells |
| US10052497B2 (en) * | 2005-07-22 | 2018-08-21 | The Board Of Trustees Of The Leland Stanford Junior University | System for optical stimulation of target cells |
| US9274099B2 (en) | 2005-07-22 | 2016-03-01 | The Board Of Trustees Of The Leland Stanford Junior University | Screening test drugs to identify their effects on cell membrane voltage-gated ion channel |
| US8926959B2 (en) * | 2005-07-22 | 2015-01-06 | The Board Of Trustees Of The Leland Stanford Junior University | System for optical stimulation of target cells |
| WO2007146046A2 (en) | 2006-06-07 | 2007-12-21 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| WO2008086470A1 (en) | 2007-01-10 | 2008-07-17 | The Board Of Trustees Of The Leland Stanford Junior University | System for optical stimulation of target cells |
| WO2008101128A1 (en) | 2007-02-14 | 2008-08-21 | The Board Of Trustees Of The Leland Stanford Junior University | System, method and applications involving identification of biological circuits such as neurological characteristics |
| WO2008106694A2 (en) | 2007-03-01 | 2008-09-04 | The Board Of Trustees Of The Leland Stanford Junior University | Systems, methods and compositions for optical stimulation of target cells |
| EP2019143A1 (en) * | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| US10434327B2 (en) | 2007-10-31 | 2019-10-08 | The Board Of Trustees Of The Leland Stanford Junior University | Implantable optical stimulators |
| US10035027B2 (en) * | 2007-10-31 | 2018-07-31 | The Board Of Trustees Of The Leland Stanford Junior University | Device and method for ultrasonic neuromodulation via stereotactic frame based technique |
| MY169771A (en) | 2008-04-23 | 2019-05-15 | Univ Leland Stanford Junior | Systems, methods and compositions for optical stimulation of target cells |
| WO2009148946A2 (en) * | 2008-05-29 | 2009-12-10 | The Board Of Trustees Of The Leland Stanford Junior University | Cell line, system and method for optical control of secondary messengers |
| EP2303405A4 (en) | 2008-06-17 | 2017-12-27 | The Board of Trustees of the Leland Stanford Junior University | Apparatus and methods for controlling cellular development |
| JP2011525130A (ja) | 2008-06-17 | 2011-09-15 | ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティ | 光伝送素子を用いた標的細胞の光刺激のための方法、システム、および装置 |
| WO2010006049A1 (en) | 2008-07-08 | 2010-01-14 | The Board Of Trustees Of The Leland Stanford Junior University | Materials and approaches for optical stimulation of the peripheral nervous system |
| NZ602416A (en) | 2008-11-14 | 2014-08-29 | Univ Leland Stanford Junior | Optically-based stimulation of target cells and modifications thereto |
| JP5879256B2 (ja) * | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
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| EP1879623A4 (en) | 2009-03-25 |
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| IL237658A0 (en) | 2015-04-30 |
| JP2017132774A (ja) | 2017-08-03 |
| DK1879623T3 (da) | 2012-12-17 |
| AU2006242129A1 (en) | 2006-11-09 |
| WO2006119341A3 (en) | 2007-06-28 |
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| JP2020050660A (ja) | 2020-04-02 |
| US10744210B2 (en) | 2020-08-18 |
| PT1879623E (pt) | 2012-12-20 |
| JP2008540435A (ja) | 2008-11-20 |
| ES2394482T3 (es) | 2013-02-01 |
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