JP7088473B2 - ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 - Google Patents
ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 Download PDFInfo
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- JP7088473B2 JP7088473B2 JP2018521617A JP2018521617A JP7088473B2 JP 7088473 B2 JP7088473 B2 JP 7088473B2 JP 2018521617 A JP2018521617 A JP 2018521617A JP 2018521617 A JP2018521617 A JP 2018521617A JP 7088473 B2 JP7088473 B2 JP 7088473B2
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- Prior art keywords
- leukodystrophy
- dendrimer
- peroxisome
- disease
- cerebral
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
- A61K31/05—Phenols
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
- A61K31/19—Carboxylic acids, e.g. valproic acid
- A61K31/192—Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
- A61K31/19—Carboxylic acids, e.g. valproic acid
- A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
- A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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- 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
- A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/56—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/59—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
- A61K47/595—Polyamides, e.g. nylon
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- A—HUMAN NECESSITIES
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- 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
- A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/56—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/59—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
- A61K47/60—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/001—Preparation for luminescence or biological staining
- A61K49/0013—Luminescence
- A61K49/0017—Fluorescence in vivo
- A61K49/0019—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules
- A61K49/0021—Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules the fluorescent group being a small organic molecule
- A61K49/0032—Methine dyes, e.g. cyanine dyes
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- A—HUMAN NECESSITIES
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- A61K49/001—Preparation for luminescence or biological staining
- A61K49/0013—Luminescence
- A61K49/0017—Fluorescence in vivo
- A61K49/005—Fluorescence in vivo characterised by the carrier molecule carrying the fluorescent agent
- A61K49/0054—Macromolecular compounds, i.e. oligomers, polymers, dendrimers
-
- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P15/00—Drugs for genital or sexual disorders; Contraceptives
- A61P15/08—Drugs for genital or sexual disorders; Contraceptives for gonadal disorders or for enhancing fertility, e.g. inducers of ovulation or of spermatogenesis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/18—Antioxidants, e.g. antiradicals
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- 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
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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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P39/00—General protective or antinoxious agents
- A61P39/06—Free radical scavengers or antioxidants
-
- 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
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- Life Sciences & Earth Sciences (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Organic Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Endocrinology (AREA)
- Diabetes (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Reproductive Health (AREA)
- Rheumatology (AREA)
- Pathology (AREA)
- Urology & Nephrology (AREA)
- Gastroenterology & Hepatology (AREA)
- Gynecology & Obstetrics (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pregnancy & Childbirth (AREA)
- Dermatology (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022087580A JP2022105767A (ja) | 2015-10-29 | 2022-05-30 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562248163P | 2015-10-29 | 2015-10-29 | |
| US62/248,163 | 2015-10-29 | ||
| PCT/US2016/059697 WO2017075580A1 (en) | 2015-10-29 | 2016-10-31 | Compositions and methods for treatment of peroxisomal disorders and leukodystrophies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022087580A Division JP2022105767A (ja) | 2015-10-29 | 2022-05-30 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018531965A JP2018531965A (ja) | 2018-11-01 |
| JP2018531965A5 JP2018531965A5 (enExample) | 2019-12-12 |
| JP7088473B2 true JP7088473B2 (ja) | 2022-06-21 |
Family
ID=57233968
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521617A Active JP7088473B2 (ja) | 2015-10-29 | 2016-10-31 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
| JP2022087580A Pending JP2022105767A (ja) | 2015-10-29 | 2022-05-30 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022087580A Pending JP2022105767A (ja) | 2015-10-29 | 2022-05-30 | ペルオキシソーム障害および白質ジストロフィーの処置のための組成物および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US20170119899A1 (enExample) |
| EP (1) | EP3368088B1 (enExample) |
| JP (2) | JP7088473B2 (enExample) |
| CN (1) | CN108430514B (enExample) |
| AU (1) | AU2016343855B2 (enExample) |
| CA (1) | CA3003589C (enExample) |
| WO (1) | WO2017075580A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6342575B2 (ja) * | 2014-08-13 | 2018-06-13 | ザ・ジョンズ・ホプキンス・ユニバーシティー | 脳腫瘍への選択的デンドリマー送達 |
| EP3615086A4 (en) | 2017-04-27 | 2021-01-06 | The Johns Hopkins University | COMPOSITIONS OF DENDRIMERS FOR USE IN ANGIOGRAPHY |
| WO2018208707A1 (en) * | 2017-05-08 | 2018-11-15 | Neurovia, Inc. | Methods and compositions for treating demyelinating diseases |
| CN111615528A (zh) | 2017-11-10 | 2020-09-01 | 约翰霍普金斯大学 | 树枝状聚合物递送系统和其使用方法 |
| US11679104B2 (en) | 2017-12-15 | 2023-06-20 | Duke University | Compositions and methods of enhancing the homing and/or engraftment of hematopoietic cells in the central nervous system |
| EP3866811A4 (en) * | 2018-10-19 | 2022-11-02 | Disarm Therapeutics, Inc. | INHIBITORS OF SARM1 IN COMBINATION WITH NAD+ OR A NAD+ PRECURSOR |
| WO2020142373A1 (en) * | 2019-01-02 | 2020-07-09 | Mayo Foundation For Medical Education And Research | Methods and materials for treating leukodystrophies |
| JP7759322B2 (ja) | 2019-12-04 | 2025-10-23 | アシュバッタ セラピューティクス, インコーポレイテッド | 眼に薬物送達するためのデンドリマー組成物および方法 |
| CN116075322A (zh) * | 2020-04-24 | 2023-05-05 | 阿什瓦塔治疗股份有限公司 | 用于治疗重度急性呼吸窘迫综合征的树枝状大分子组合物和方法 |
| PH12023550133A1 (en) * | 2020-07-17 | 2024-06-24 | Ashvattha Therapeutics Inc | Dendrimer compositions and methods for drug delivery to injured kidney |
| WO2023081750A1 (en) * | 2021-11-04 | 2023-05-11 | The Children's Hospital Of Philadelphia | Compositions and methods for treating metachromatic leukodystrophy disease and related disorders |
| WO2024081687A2 (en) * | 2022-10-12 | 2024-04-18 | The Johns Hopkins University | Method of treating lbsl by enhancing dars2 expression |
| CN116082153A (zh) * | 2022-12-09 | 2023-05-09 | 长沙贝塔医药科技有限公司 | 一种丙烯酸酯类衍生物-13c的制备方法 |
| EP4410284A1 (en) * | 2023-02-02 | 2024-08-07 | Apteeus | Compound and method for the treatment of zellweger spectrum disorder |
| CN117643581A (zh) * | 2023-11-29 | 2024-03-05 | 徐州医科大学 | 白藜芦醇在制备激活sirt1基因的药物中的应用 |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2010540664A (ja) | 2007-10-05 | 2010-12-24 | ウェイン ステート ユニバーシティー | 合成物の持続的な放出のためのデンドリマー |
| US20120003155A1 (en) | 2009-06-15 | 2012-01-05 | National Institutes Of Health | Dendrimer based nanodevices for therapeutic and imaging purposes |
| WO2015027068A1 (en) | 2013-08-21 | 2015-02-26 | Concordia University | Dendrimer-resveratrol complex |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2212047C (en) | 1995-02-07 | 2010-11-16 | Saul W. Brusilow | Triglycerides and ethyl esters of phenylalkanoic acid and phenylalkenoic acid useful in treatment of various disorders |
| CA2344717A1 (en) * | 1998-09-28 | 2000-04-06 | The Johns Hopkins University | Adrenoleukodystrophy treatments and drug screening |
| US8148356B2 (en) | 2005-08-24 | 2012-04-03 | Cumberland Pharmaceuticals, Inc. | Acetylcysteine composition and uses therefor |
| TW200817369A (en) * | 2006-05-22 | 2008-04-16 | Elan Pharm Inc | Preparation of polymer conjugates of therapeutic, agricultural and food additive compounds |
| US8642012B2 (en) | 2008-04-29 | 2014-02-04 | Hyperion Therapeutics, Inc. | Methods of treatment using ammonia-scavenging drugs |
| WO2009142754A1 (en) * | 2008-05-22 | 2009-11-26 | Goverment Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Dendritic conjugates and methods of use |
| WO2012012640A1 (en) | 2010-07-21 | 2012-01-26 | Cumberland Pharmaceuticals, Inc. | Acetycysteine compositions and methods of use thereof |
| BR112014007357B1 (pt) | 2011-09-30 | 2022-12-06 | Immedica Pharma Ab | Método para determinar o ajuste da dosagem de um fármaco de varredura de nitrogênio |
| JP2016517883A (ja) * | 2013-05-03 | 2016-06-20 | オレゴン ヘルス アンド サイエンス ユニバーシティー | X連鎖副腎白質ジストロフィーの処置におけるソベチロムの使用 |
| US20160122406A1 (en) * | 2013-06-07 | 2016-05-05 | The Administrators Of The Tulane Educational Fund | Analogs of pituitary adenylate cyclase-activating polypeptide (pacap) and methods for their use |
| US20170232120A1 (en) * | 2014-08-13 | 2017-08-17 | The Johns Hopkins University | Dendrimer compositions and use in treatment of neurological and cns disorders |
-
2016
- 2016-10-31 WO PCT/US2016/059697 patent/WO2017075580A1/en not_active Ceased
- 2016-10-31 CA CA3003589A patent/CA3003589C/en active Active
- 2016-10-31 AU AU2016343855A patent/AU2016343855B2/en active Active
- 2016-10-31 CN CN201680073817.9A patent/CN108430514B/zh active Active
- 2016-10-31 EP EP16791282.3A patent/EP3368088B1/en active Active
- 2016-10-31 JP JP2018521617A patent/JP7088473B2/ja active Active
- 2016-10-31 US US15/339,486 patent/US20170119899A1/en not_active Abandoned
-
2021
- 2021-09-20 US US17/479,400 patent/US20220071923A1/en not_active Abandoned
-
2022
- 2022-05-30 JP JP2022087580A patent/JP2022105767A/ja active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2010540664A (ja) | 2007-10-05 | 2010-12-24 | ウェイン ステート ユニバーシティー | 合成物の持続的な放出のためのデンドリマー |
| US20120003155A1 (en) | 2009-06-15 | 2012-01-05 | National Institutes Of Health | Dendrimer based nanodevices for therapeutic and imaging purposes |
| WO2015027068A1 (en) | 2013-08-21 | 2015-02-26 | Concordia University | Dendrimer-resveratrol complex |
Non-Patent Citations (7)
| Title |
|---|
| ACS Nano,2014年02月05日,Vol. 8, No. 3,pp. 2134-2147 |
| Brain Pathology,2010年,Vol. 20,pp. 845-856 |
| Journal of Controlled Release,2015年,Vol. 214,pp. 112-120 |
| Journal of Neuropathology and Experimental Neurology,2000年,Vol. 59, No. 2,pp. 89-102 |
| Nature Medicine,1998年,Vol. 4, No. 11,pp. 1261-1268 |
| The Journal of Cell Biology,2005年,Vol. 169, No. 1,pp.93-104 |
| メルクマニュアル 第18版 日本語版,初版第3刷,2007年,pp. 2626-2627,(最終査読2016年1月のMSDマニュアルプロフェッショナル版のウェブページも添付した。) |
Also Published As
| Publication number | Publication date |
|---|---|
| CA3003589A1 (en) | 2017-05-04 |
| AU2016343855B2 (en) | 2019-10-03 |
| US20220071923A1 (en) | 2022-03-10 |
| WO2017075580A1 (en) | 2017-05-04 |
| EP3368088C0 (en) | 2023-06-07 |
| CA3003589C (en) | 2021-12-07 |
| AU2016343855A1 (en) | 2018-05-10 |
| JP2022105767A (ja) | 2022-07-14 |
| US20170119899A1 (en) | 2017-05-04 |
| JP2018531965A (ja) | 2018-11-01 |
| CN108430514B (zh) | 2023-11-03 |
| EP3368088B1 (en) | 2023-06-07 |
| EP3368088A1 (en) | 2018-09-05 |
| CN108430514A (zh) | 2018-08-21 |
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