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 PDFInfo
- Publication number
- 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
- Authority
- CN
- China
- Prior art keywords
- subpial
- delivery system
- aav9
- gene delivery
- spinal cord
- 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.)
- Active
Links
Images
Classifications
-
- 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
- C12N15/11—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
-
- 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
- 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
- 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
- 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
- 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
-
- 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
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- 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
-
- 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
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- A61P25/14—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
- A61P25/20—Hypnotics; Sedatives
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- A61M2210/00—Anatomical parts of the body
- A61M2210/10—Trunk
- A61M2210/1003—Spinal column
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Engineering & Computer Science (AREA)
- Neurosurgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Virology (AREA)
- Psychology (AREA)
- Anesthesiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Inorganic Chemistry (AREA)
- Hematology (AREA)
- Heart & Thoracic Surgery (AREA)
- Pulmonology (AREA)
- Immunology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN107427666A CN107427666A (zh) | 2017-12-01 |
| CN107427666B true CN107427666B (zh) | 2022-11-04 |
Family
ID=56544138
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201580078566.9A Active CN107427666B (zh) | 2015-01-30 | 2015-12-15 | 脊髓软膜下基因递送系统 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20180008727A1 (enExample) |
| EP (1) | EP3250279B1 (enExample) |
| JP (5) | JP2018506530A (enExample) |
| CN (1) | CN107427666B (enExample) |
| CA (1) | CA2975447C (enExample) |
| WO (1) | WO2016122791A1 (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| US10688285B2 (en) | 2015-01-30 | 2020-06-23 | The Regents Of The University Of California | Spinal subpial gene delivery system |
| JP6949867B2 (ja) | 2016-03-28 | 2021-10-13 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California | 神経の過剰興奮を治療するための方法および組成物 |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| AU2018327253B2 (en) * | 2017-09-08 | 2024-05-16 | The Regents Of The University Of California | Method and composition for treating neuropathic pain |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| MX2020012077A (es) | 2018-05-15 | 2021-03-09 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de parkinson. |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| JP7373226B2 (ja) * | 2019-01-10 | 2023-11-02 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 軟膜下送達システムおよび使用方法 |
| USD963164S1 (en) | 2020-01-09 | 2022-09-06 | The Regents Of The University Of California | Surgical needle |
| AU2023389530A1 (en) | 2022-12-05 | 2025-07-03 | Eg 427 | Viral vector encoding gad for treating spasticity |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101528922A (zh) * | 2006-10-03 | 2009-09-09 | 建新公司 | 肌萎缩性侧索硬化症和其它脊髓病症的基因治疗 |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5292722A (en) * | 1992-11-06 | 1994-03-08 | Brigham And Women's Hospital | Intravenous solution that diminishes body protein loss |
| FR2705361B1 (fr) | 1993-05-18 | 1995-08-04 | Centre Nat Rech Scient | Vecteurs viraux et utilisation en thérapie génique. |
| JP3532566B2 (ja) | 1993-06-24 | 2004-05-31 | エル. グラハム,フランク | 遺伝子治療のためのアデノウイルスベクター |
| ES2310924T3 (es) | 1993-07-13 | 2009-01-16 | Centelion | Vectores adenovirales defectivos y utilizacion en terapia genica. |
| ATE272123T1 (de) | 1993-11-09 | 2004-08-15 | Ohio Med College | Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren |
| EP0733103B1 (en) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| WO1995016772A1 (en) | 1993-12-14 | 1995-06-22 | Cornell Research Foundation, Inc. | Adenovirus gene expression system |
| FR2716893B1 (fr) | 1994-03-03 | 1996-04-12 | Rhone Poulenc Rorer Sa | Virus recombinants, leur préparation et leur utilisation thérapeutique. |
| AUPM452094A0 (en) | 1994-03-17 | 1994-04-14 | University Of Queensland, The | Waste treatment plant and process |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| US20030032614A1 (en) * | 1995-04-13 | 2003-02-13 | Mcmichael John | Method of treating respiratory distress using synthetic polynucleic acids |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US20020007145A1 (en) * | 1998-10-23 | 2002-01-17 | Timothy Stivland | Catheter having improved bonding region |
| US7125856B1 (en) * | 1999-04-15 | 2006-10-24 | St. Elizabeth's Medical Center Of Boston, Inc. | Angiogenic growth factors for treatment of peripheral neuropathy |
| ATE330001T1 (de) * | 2000-04-12 | 2006-07-15 | Philadelphia Children Hospital | Therapeutische verwendung mesenchymaler stromazellen |
| US7011647B2 (en) * | 2001-07-13 | 2006-03-14 | Scimed Life Systems, Inc. | Introducer sheath |
| US20070213292A1 (en) * | 2005-08-10 | 2007-09-13 | The Rockefeller University | Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof |
| AU2008261668C1 (en) * | 2007-06-11 | 2014-01-16 | Edge Therapeutics Inc. | A drug delivery system for the prevention of cerebral vasospasm |
| WO2009091514A2 (en) * | 2008-01-14 | 2009-07-23 | I-V Access Technology, Inc. | An apparatus for peripheral vascular access |
| WO2010071832A1 (en) * | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| CN102438978B (zh) * | 2009-03-20 | 2017-02-22 | Clsn实验室股份有限公司 | 聚胺衍生物 |
| CN102666856B (zh) | 2009-11-08 | 2016-04-06 | 夸克制药公司 | 定向至RhoA靶基因的双链RNA化合物在制造治疗神经性疼痛的药物中的用途 |
| CA2819635A1 (en) * | 2010-12-01 | 2012-06-07 | Spinal Modulation, Inc. | Directed delivery of agents to neural anatomy |
| JP6091435B2 (ja) | 2011-02-22 | 2017-03-08 | カリフォルニア インスティチュート オブ テクノロジー | アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達 |
| CN107982548A (zh) * | 2012-02-07 | 2018-05-04 | 全球生物疗法有限公司 | 核酸输送的区室化方法及其组合物和应用 |
| CA2896442A1 (en) * | 2012-09-24 | 2014-03-27 | The Regents Of The University Of California | Spinal cord pulsation-cancelation injection system |
| GB201308917D0 (en) * | 2013-05-17 | 2013-07-03 | Renishaw Plc | Delivery |
-
2015
- 2015-12-15 EP EP15880645.5A patent/EP3250279B1/en active Active
- 2015-12-15 CN CN201580078566.9A patent/CN107427666B/zh active Active
- 2015-12-15 US US15/544,973 patent/US20180008727A1/en not_active Abandoned
- 2015-12-15 JP JP2017540569A patent/JP2018506530A/ja active Pending
- 2015-12-15 CA CA2975447A patent/CA2975447C/en active Active
- 2015-12-15 WO PCT/US2015/065704 patent/WO2016122791A1/en not_active Ceased
-
2019
- 2019-10-03 JP JP2019182624A patent/JP2020019796A/ja not_active Withdrawn
-
2021
- 2021-07-29 JP JP2021124373A patent/JP2021181456A/ja active Pending
-
2023
- 2023-03-09 JP JP2023036473A patent/JP2023060308A/ja active Pending
-
2024
- 2024-11-15 JP JP2024199400A patent/JP2025015673A/ja active Pending
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101528922A (zh) * | 2006-10-03 | 2009-09-09 | 建新公司 | 肌萎缩性侧索硬化症和其它脊髓病症的基因治疗 |
Also Published As
| Publication number | Publication date |
|---|---|
| HK1247867A1 (zh) | 2018-10-05 |
| CA2975447C (en) | 2021-02-23 |
| EP3250279A4 (en) | 2018-06-20 |
| JP2018506530A (ja) | 2018-03-08 |
| EP3250279B1 (en) | 2020-08-19 |
| JP2020019796A (ja) | 2020-02-06 |
| WO2016122791A1 (en) | 2016-08-04 |
| EP3250279A1 (en) | 2017-12-06 |
| CA2975447A1 (en) | 2016-08-04 |
| JP2025015673A (ja) | 2025-01-30 |
| JP2021181456A (ja) | 2021-11-25 |
| JP2023060308A (ja) | 2023-04-27 |
| CN107427666A (zh) | 2017-12-01 |
| US20180008727A1 (en) | 2018-01-11 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CN107427666B (zh) | 脊髓软膜下基因递送系统 | |
| JP2018506530A5 (enExample) | ||
| EP3132042B1 (en) | A method of treating peripheral neuropathies and motor neuron diseases | |
| Jacques et al. | AAV8gfp preferentially targets large diameter dorsal root ganglion neurones after both intra-dorsal root ganglion and intrathecal injection | |
| JP2021521152A (ja) | 抑制性ニューロンにおける電位開口型ナトリウムチャネルのレスキュー | |
| EP3254702B1 (en) | Aav/xbp1s-ha virus, gene therapy method and use thereof in the optimisation and improvement of learning, memory and cognitive capacities | |
| CN108699565A (zh) | 用于定向腺相关病毒(aav)的靶向肽 | |
| CN108347932A (zh) | 用于治疗遗传性眼病的方法和组合物 | |
| US10688285B2 (en) | Spinal subpial gene delivery system | |
| JP2020535804A (ja) | Sod1二重発現ベクターおよびその使用 | |
| CA3133455A1 (en) | Vector and method for treating angelman syndrome | |
| JP7235676B2 (ja) | 結節性硬化症の遺伝子治療 | |
| US11434501B2 (en) | Sprr1A as a genetic target for treating neurodegenerative diseases | |
| US20230070049A1 (en) | Microrna-7 compositions for promoting functional recovery following spinal cord injury and methods of use thereof | |
| US11707535B2 (en) | Method and composition for treating neuropathic pain | |
| HK1247867B (en) | Spinal subpial gene delivery system | |
| Lowenstein et al. | Gene therapy for neurological disorders | |
| WO2025250454A1 (en) | Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo | |
| Mason | SRSF1-mediated gene therapy as a therapeutic approach for C9ORF72-related amyotrophic lateral sclerosis and fronto-temporal dementia (ALS-FTD) | |
| US20210381002A1 (en) | Gene therapy for oxidative stress | |
| Aubourg | Pediatric Neurology Part III: Chapter 191. Gene therapy for disorders of the central nervous system | |
| Wu | Delivery of Helper-Dependent Adenoviral Vectors to the Subretinal Space of Mice | |
| Pietersz et al. | PhP. B-enhanced AAV expression following systemic delivery or direct brain administration | |
| BR112017017867B1 (pt) | Proteína do capsídeo do vírus adenoassociado modificado (aav), sequência de ácido nucleico, partícula de vetor viral, composição farmacêutica, bem como usos do vetor viral |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PB01 | Publication | ||
| PB01 | Publication | ||
| 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: 1247867 Country of ref document: HK |
|
| GR01 | Patent grant | ||
| GR01 | Patent grant |