CR20150216A - Composiciones y métodos para tratar proteinopatías - Google Patents
Composiciones y métodos para tratar proteinopatíasInfo
- Publication number
- CR20150216A CR20150216A CR20150216A CR20150216A CR20150216A CR 20150216 A CR20150216 A CR 20150216A CR 20150216 A CR20150216 A CR 20150216A CR 20150216 A CR20150216 A CR 20150216A CR 20150216 A CR20150216 A CR 20150216A
- Authority
- CR
- Costa Rica
- Prior art keywords
- methods
- proteinopathies
- treat
- compositions
- mammal
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
- 241000124008 Mammalia Species 0.000 abstract 2
- 102000004547 Glucosylceramidase Human genes 0.000 abstract 1
- 108010017544 Glucosylceramidase Proteins 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 230000001537 neural effect Effects 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
-
- 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
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61P25/14—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
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
-
- 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
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Mycology (AREA)
- Dermatology (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261722434P | 2012-11-05 | 2012-11-05 | |
| PCT/US2013/068242 WO2014071282A1 (en) | 2012-11-05 | 2013-11-04 | Compositions and methods for treating proteinopathies |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CR20150216A true CR20150216A (es) | 2015-05-29 |
Family
ID=49578599
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CR20150216A CR20150216A (es) | 2012-11-05 | 2015-04-28 | Composiciones y métodos para tratar proteinopatías |
Country Status (22)
| Country | Link |
|---|---|
| US (1) | US20150284472A1 (OSRAM) |
| EP (1) | EP2914281A1 (OSRAM) |
| JP (1) | JP2016503405A (OSRAM) |
| KR (1) | KR20150079751A (OSRAM) |
| CN (1) | CN104902923A (OSRAM) |
| AR (1) | AR093355A1 (OSRAM) |
| AU (1) | AU2013337354A1 (OSRAM) |
| BR (1) | BR112015009746A2 (OSRAM) |
| CA (1) | CA2889990A1 (OSRAM) |
| CL (1) | CL2015001157A1 (OSRAM) |
| CR (1) | CR20150216A (OSRAM) |
| EA (1) | EA201590880A1 (OSRAM) |
| HK (1) | HK1214521A1 (OSRAM) |
| IL (1) | IL238416A0 (OSRAM) |
| MA (1) | MA38144A1 (OSRAM) |
| MX (1) | MX2015005722A (OSRAM) |
| PH (1) | PH12015500879A1 (OSRAM) |
| SG (1) | SG11201502989XA (OSRAM) |
| TN (1) | TN2015000171A1 (OSRAM) |
| TW (1) | TW201427695A (OSRAM) |
| WO (1) | WO2014071282A1 (OSRAM) |
| ZA (1) | ZA201502618B (OSRAM) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2963910T3 (es) | 2008-06-26 | 2024-04-03 | Zevra Denmark As | Uso de Hsp70 como regulador de la actividad enzimática |
| PT2646044T (pt) | 2010-11-30 | 2019-11-12 | Orphazyme As | Métodos para aumentar a atividade intracelular de hsp70 |
| HUE054957T2 (hu) | 2014-09-15 | 2021-10-28 | Orphazyme As | Arimoklomol készítése |
| EP3292206B8 (en) * | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| SG11201901265SA (en) * | 2015-07-21 | 2019-03-28 | Univ Jefferson | Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways |
| MX2018008743A (es) * | 2016-01-21 | 2019-01-30 | Protein Dynamic Solutions Llc | Método y sistema para el análisis de datos espectrales. |
| US10898476B2 (en) | 2016-04-13 | 2021-01-26 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| RU2021117465A (ru) * | 2016-04-29 | 2021-07-22 | Орфазим А/С | Аримокломол для лечения ассоциированных с глюкоцереброзидазой нарушений |
| EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
| AU2017306558B2 (en) * | 2016-08-03 | 2024-09-19 | University Of South Florida | Reelin compositions for treatment of neurological disorders |
| CN111465691A (zh) | 2017-10-03 | 2020-07-28 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| JP7254815B2 (ja) * | 2017-10-03 | 2023-04-10 | プリベイル セラピューティクス,インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| CN111819281B (zh) * | 2017-10-23 | 2024-12-31 | 普利维尔治疗公司 | 用于神经变性疾病的基因疗法 |
| KR20240171186A (ko) * | 2017-10-26 | 2024-12-06 | 다케다 야쿠힌 고교 가부시키가이샤 | 글루코세레브로시다제 및 이소파고민을 포함하는 제제 |
| WO2019175883A1 (en) * | 2018-03-14 | 2019-09-19 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd | Detection of phospho-serine 129 alpha-synuclein in blood cells as a biomarker for synucleinopathies |
| AU2020273182B2 (en) | 2019-04-10 | 2025-11-20 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN114026115A (zh) * | 2019-04-10 | 2022-02-08 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
| EP3986412A4 (en) | 2019-06-21 | 2023-11-15 | The Broad Institute, Inc. | AGENTS TO REVERSE TOXIC PROTEINOPATHIES |
| EP4192961A2 (en) | 2020-08-06 | 2023-06-14 | Fundacion para la Investigacion Medica Aplicada | Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy |
| EP4229419A2 (en) * | 2020-10-14 | 2023-08-23 | Denali Therapeutics Inc. | Methods for treating and monitoring frontotemporal dementia |
| IL303026A (en) | 2020-11-19 | 2023-07-01 | Zevra Denmark As | Processes for preparing arimoclomol citrate and intermediates thereof |
| CN112569354B (zh) * | 2020-12-29 | 2022-06-10 | 四川大学华西医院 | tau蛋白及其基因作为药物靶点在制备治疗糖尿病药物中的应用 |
| JP2024546887A (ja) * | 2021-12-17 | 2024-12-26 | ジェネンテック, インコーポレイテッド | オリゴヌクレオチドgbaアゴニスト |
| WO2023111335A1 (en) * | 2021-12-17 | 2023-06-22 | F. Hoffmann-La Roche Ag | Oligonucleotides capable of increasing glucocerebrosidase expression |
Family Cites Families (33)
| Publication number | Priority date | Publication date | Assignee | Title |
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| US4816567A (en) | 1983-04-08 | 1989-03-28 | Genentech, Inc. | Recombinant immunoglobin preparations |
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| US4676980A (en) | 1985-09-23 | 1987-06-30 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Target specific cross-linked heteroantibodies |
| US5225539A (en) | 1986-03-27 | 1993-07-06 | Medical Research Council | Recombinant altered antibodies and methods of making altered antibodies |
| US5892019A (en) | 1987-07-15 | 1999-04-06 | The United States Of America, As Represented By The Department Of Health And Human Services | Production of a single-gene-encoded immunoglobulin |
| GB8823869D0 (en) | 1988-10-12 | 1988-11-16 | Medical Res Council | Production of antibodies |
| US6172197B1 (en) | 1991-07-10 | 2001-01-09 | Medical Research Council | Methods for producing members of specific binding pairs |
| GB9015198D0 (en) | 1990-07-10 | 1990-08-29 | Brien Caroline J O | Binding substance |
| US5661016A (en) | 1990-08-29 | 1997-08-26 | Genpharm International Inc. | Transgenic non-human animals capable of producing heterologous antibodies of various isotypes |
| JP2938569B2 (ja) | 1990-08-29 | 1999-08-23 | ジェンファーム インターナショナル,インコーポレイティド | 異種免疫グロブリンを作る方法及びトランスジェニックマウス |
| US5625126A (en) | 1990-08-29 | 1997-04-29 | Genpharm International, Inc. | Transgenic non-human animals for producing heterologous antibodies |
| US5545806A (en) | 1990-08-29 | 1996-08-13 | Genpharm International, Inc. | Ransgenic non-human animals for producing heterologous antibodies |
| US5633425A (en) | 1990-08-29 | 1997-05-27 | Genpharm International, Inc. | Transgenic non-human animals capable of producing heterologous antibodies |
| WO1992009690A2 (en) | 1990-12-03 | 1992-06-11 | Genentech, Inc. | Enrichment method for variant proteins with altered binding properties |
| WO1993011236A1 (en) | 1991-12-02 | 1993-06-10 | Medical Research Council | Production of anti-self antibodies from antibody segment repertoires and displayed on phage |
| AU687829B2 (en) | 1993-06-24 | 1998-03-05 | Advec, Inc. | Adenovirus vectors for gene therapy |
| JP3875990B2 (ja) | 1993-10-25 | 2007-01-31 | カンジ,インコーポレイテッド | 組換えアデノウイルスベクターおよび使用方法 |
| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
| US7264963B1 (en) | 1995-08-18 | 2007-09-04 | Morphosys Ag | Protein(poly)peptide libraries |
| ES2176484T3 (es) | 1995-08-18 | 2002-12-01 | Morphosys Ag | Bancos de proteinas/(poli)peptidos. |
| US6924123B2 (en) | 1996-10-29 | 2005-08-02 | Oxford Biomedica (Uk) Limited | Lentiviral LTR-deleted vector |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| ATE482275T1 (de) | 1999-07-02 | 2010-10-15 | Morphosys Ag | Erzeugung spezifischer bindungspartner die an von genomischen dns-fragmenten oder ests kodierten (poly)peptiden binden |
| ES2308989T3 (es) | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios. |
| AU6972301A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Duplexed parvovirus vectors |
| WO2001097829A2 (en) | 2000-06-19 | 2001-12-27 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
| JP5303458B2 (ja) * | 2006-06-23 | 2013-10-02 | アミークス セラピューティックス インコーポレイテッド | β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法 |
| HUE027278T2 (en) * | 2007-05-16 | 2016-11-28 | Brigham & Womens Hospital Inc | Treatment of synucleinopathies |
| WO2012177997A1 (en) | 2011-06-22 | 2012-12-27 | The General Hospital Corporation | Treatment of proteinopathies |
| AU2013240187A1 (en) | 2012-03-28 | 2014-10-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Salicylic acid derivatives useful as glucocerebrosidase activators |
-
2013
- 2013-11-04 BR BR112015009746A patent/BR112015009746A2/pt not_active IP Right Cessation
- 2013-11-04 HK HK16102536.9A patent/HK1214521A1/zh unknown
- 2013-11-04 CN CN201380069582.2A patent/CN104902923A/zh active Pending
- 2013-11-04 JP JP2015540845A patent/JP2016503405A/ja not_active Abandoned
- 2013-11-04 CA CA2889990A patent/CA2889990A1/en not_active Abandoned
- 2013-11-04 AU AU2013337354A patent/AU2013337354A1/en not_active Abandoned
- 2013-11-04 US US14/440,818 patent/US20150284472A1/en not_active Abandoned
- 2013-11-04 KR KR1020157013541A patent/KR20150079751A/ko not_active Withdrawn
- 2013-11-04 WO PCT/US2013/068242 patent/WO2014071282A1/en not_active Ceased
- 2013-11-04 EP EP13789690.8A patent/EP2914281A1/en not_active Withdrawn
- 2013-11-04 SG SG11201502989XA patent/SG11201502989XA/en unknown
- 2013-11-04 TW TW102139891A patent/TW201427695A/zh unknown
- 2013-11-04 MA MA38144A patent/MA38144A1/fr unknown
- 2013-11-04 EA EA201590880A patent/EA201590880A1/ru unknown
- 2013-11-04 MX MX2015005722A patent/MX2015005722A/es unknown
- 2013-11-05 AR ARP130104033A patent/AR093355A1/es unknown
-
2015
- 2015-04-17 ZA ZA2015/02618A patent/ZA201502618B/en unknown
- 2015-04-20 PH PH12015500879A patent/PH12015500879A1/en unknown
- 2015-04-21 IL IL238416A patent/IL238416A0/en unknown
- 2015-04-28 CR CR20150216A patent/CR20150216A/es unknown
- 2015-04-30 CL CL2015001157A patent/CL2015001157A1/es unknown
- 2015-05-04 TN TNP2015000171A patent/TN2015000171A1/fr unknown
Also Published As
| Publication number | Publication date |
|---|---|
| BR112015009746A2 (pt) | 2017-08-15 |
| TN2015000171A1 (fr) | 2016-10-03 |
| AR093355A1 (es) | 2015-06-03 |
| MA38144A1 (fr) | 2018-08-31 |
| TW201427695A (zh) | 2014-07-16 |
| US20150284472A1 (en) | 2015-10-08 |
| AU2013337354A1 (en) | 2015-05-21 |
| CN104902923A (zh) | 2015-09-09 |
| JP2016503405A (ja) | 2016-02-04 |
| HK1214521A1 (zh) | 2016-07-29 |
| EA201590880A1 (ru) | 2015-09-30 |
| WO2014071282A1 (en) | 2014-05-08 |
| ZA201502618B (en) | 2016-01-27 |
| PH12015500879A1 (en) | 2015-06-29 |
| EP2914281A1 (en) | 2015-09-09 |
| MX2015005722A (es) | 2016-01-12 |
| CL2015001157A1 (es) | 2015-10-16 |
| CA2889990A1 (en) | 2014-05-08 |
| SG11201502989XA (en) | 2015-05-28 |
| KR20150079751A (ko) | 2015-07-08 |
| IL238416A0 (en) | 2015-06-30 |
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