HRP20171165T1 - Genska terapija za lizosomske bolesti nakupljanja - Google Patents
Genska terapija za lizosomske bolesti nakupljanja Download PDFInfo
- Publication number
- HRP20171165T1 HRP20171165T1 HRP20171165TT HRP20171165T HRP20171165T1 HR P20171165 T1 HRP20171165 T1 HR P20171165T1 HR P20171165T T HRP20171165T T HR P20171165TT HR P20171165 T HRP20171165 T HR P20171165T HR P20171165 T1 HRP20171165 T1 HR P20171165T1
- Authority
- HR
- Croatia
- Prior art keywords
- aav vector
- recombinant aav
- use according
- vector
- brain
- Prior art date
Links
- 208000015439 Lysosomal storage disease Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 239000013607 AAV vector Substances 0.000 claims 9
- 238000000034 method Methods 0.000 claims 5
- 210000004556 brain Anatomy 0.000 claims 4
- 239000013598 vector Substances 0.000 claims 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 208000035343 Infantile neurovisceral acid sphingomyelinase deficiency Diseases 0.000 claims 2
- 201000000794 Niemann-Pick disease type A Diseases 0.000 claims 2
- 108700019146 Transgenes Proteins 0.000 claims 2
- 208000007824 Type A Niemann-Pick Disease Diseases 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 208000035374 Chronic visceral acid sphingomyelinase deficiency Diseases 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- 201000000791 Niemann-Pick disease type B Diseases 0.000 claims 1
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 1
- 208000008291 Type B Niemann-Pick Disease Diseases 0.000 claims 1
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 claims 1
- 210000003169 central nervous system Anatomy 0.000 claims 1
- 210000004055 fourth ventricle Anatomy 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 210000003140 lateral ventricle Anatomy 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 210000001835 viscera Anatomy 0.000 claims 1
Classifications
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- 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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Claims (6)
1. Rekombinantni AAV vektor koji sadrži transgen kodira kiselinu sfingomijelinazu za upotrebu u postupku liječenja Niemann–Pickove bolesti tip A ili Niemann–Pickove bolesti tip B kod subjekta, naznačen time da navedeni rekombinantni AAV vektor je AAV5 vektor i pri čemu postupak sadrži davanje vektora u barem jednu ventrikulu mozga od subjekta, pri čemu se navedeni transgen eksprimira i eksprimirani proizvod proteina se dovodi u središnji živčani sustav, te proizvoljno, u visceralne organe.
2. Rekombinantni AAV vektor za uporabu prema zahtjevu 1, naznačen time da navedeni rekombinantni AAV vektor je AAV vektor koji sadrži AAV5 ovojnicu i AAV2 genom.
3. Rekombinantni AAV vektor za uporabu prema zahtjevu 1 ili zahtjevu 2, naznačen time da postupak sadrži davanje vektora izravnim ubrizgavanjem u: (i) ventrikulu mozga; (ii) lateralnu ventrikulu mozga; ili (iii) četvrtu ventrikulu mozga.
4. Rekombinantni AAV vektor za uporabu prema bilo kojem od zahtjeva 1 do 3, naznačen time da subjekt je sisavac.
5. Rekombinantni AAV vektor za uporabu prema zahtjevu 4, naznačen time da subjekt je ljudski pacijent.
6. Rekombinantni AAV vektor za uporabu prema bilo kojem od zahtjeva 1 do 5, naznačen time da navedeni postupak je postupak liječenja Niemann–Pickove bolesti tip A.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US92494707P | 2007-06-06 | 2007-06-06 | |
PCT/US2008/065481 WO2008154198A1 (en) | 2007-06-06 | 2008-06-02 | Gene therapy for lysosomal storage diseases |
EP08756596.6A EP2158322B1 (en) | 2007-06-06 | 2008-06-02 | Gene therapy for lysosomal storage diseases |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20171165T1 true HRP20171165T1 (hr) | 2017-12-15 |
Family
ID=40130114
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20171165TT HRP20171165T1 (hr) | 2007-06-06 | 2017-07-31 | Genska terapija za lizosomske bolesti nakupljanja |
Country Status (14)
Country | Link |
---|---|
US (2) | US8796236B2 (hr) |
EP (2) | EP2158322B1 (hr) |
AR (1) | AR070000A1 (hr) |
CY (1) | CY1119295T1 (hr) |
DK (1) | DK2158322T3 (hr) |
ES (2) | ES2905616T3 (hr) |
HR (1) | HRP20171165T1 (hr) |
HU (1) | HUE035779T2 (hr) |
IL (3) | IL202522A (hr) |
LT (1) | LT2158322T (hr) |
PL (2) | PL3252161T3 (hr) |
PT (2) | PT3252161T (hr) |
SI (1) | SI2158322T1 (hr) |
WO (1) | WO2008154198A1 (hr) |
Families Citing this family (34)
Publication number | Priority date | Publication date | Assignee | Title |
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AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
DK1988823T3 (en) | 2006-02-09 | 2018-12-03 | Genzyme Corp | SLOW INTRAVENTRICULAR ADMINISTRATION |
PL3252161T3 (pl) | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US8242086B2 (en) * | 2008-11-12 | 2012-08-14 | Duke University | Methods and compositions for treating disorders caused by a deficiency in a gene product of a CLN gene |
DK2424571T3 (da) | 2009-04-30 | 2020-05-04 | Ospedale San Raffaele Srl | Genvektor |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
CA3049237C (en) | 2010-04-23 | 2024-06-11 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
CA3050894C (en) | 2010-04-23 | 2022-10-18 | University Of Massachusetts | Multicistronic expression constructs |
EP3546584B1 (en) | 2011-03-31 | 2024-02-14 | University of Iowa Research Foundation | Compositions for treating lysosmal storage disease (lsd) |
US20130090374A1 (en) * | 2011-04-20 | 2013-04-11 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis |
US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
PL2751279T3 (pl) | 2011-08-31 | 2018-03-30 | St. Jude Children's Research Hospital | Sposoby i kompozycje do wykrywania poziomu aktywności lizosomalnej egzocytozy i sposoby zastosowania |
BR112015028605A8 (pt) * | 2013-05-15 | 2019-12-24 | Univ Minnesota | uso de uma composição e uso de um imunossupressor e uma composição |
EP3039129B1 (en) * | 2013-08-30 | 2018-06-27 | Amgen Inc. | High titer recombinant aav vector production in adherent and suspension cells |
AU2015335923B2 (en) | 2014-10-21 | 2021-04-29 | University Of Massachusetts | Recombinant AAV variants and uses thereof |
BR112017009497A2 (pt) | 2014-11-05 | 2018-02-06 | Voyager Therapeutics, Inc. | polinucleotídeos de aadc para o tratamento da doença de parkinson |
EP3285788A4 (en) * | 2015-04-23 | 2018-12-05 | University of Massachusetts | Modulation of aav vector transgene expression |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
AU2016372035B2 (en) * | 2015-12-15 | 2023-03-30 | Genzyme Corporation | Adeno-associated viral vectors for treating mucolipidosis type ii |
PT3405215T (pt) * | 2016-01-19 | 2022-09-20 | Univ California | Métodos para o tratamento da doença de danon e outros transtornos da autofagia |
AU2017250298B2 (en) | 2016-04-15 | 2024-10-03 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
WO2018071831A1 (en) | 2016-10-13 | 2018-04-19 | University Of Massachusetts | Aav capsid designs |
US11219695B2 (en) | 2016-10-20 | 2022-01-11 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of Fabry disease |
EP3534892A4 (en) * | 2016-11-04 | 2020-05-27 | The Children's Hospital of Philadelphia | GENE TRANSFER COMPOSITIONS, METHODS AND USE FOR TREATING NEURODEGENERATIVE DISEASES |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
EP3642345A1 (en) | 2017-06-20 | 2020-04-29 | The U.S.A. As Represented By The Secretary, Department Of Health And Human Services | Codon-optimized human npc1 genes for the treatment of niemann-pick type c1 deficiency and related conditions |
AU2018335752A1 (en) | 2017-09-22 | 2020-03-12 | Christian HINDERER | Gene therapy for treating Mucopolysaccharidosis type ii |
US20210228739A1 (en) * | 2018-05-15 | 2021-07-29 | University Of Massachusetts | Raav vectors encoding of lysosomal beta-galactosidase (glb1) and cathepsin a |
EP3820537A4 (en) | 2018-07-12 | 2022-04-13 | Spacecraft Seven, LLC | GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE |
MX2021010266A (es) * | 2019-02-26 | 2021-09-23 | Univ Pennsylvania | Composiciones utiles en el tratamiento de la enfermedad de krabbe. |
BR112021020421A2 (pt) * | 2019-04-12 | 2021-12-21 | Encoded Therapeutics Inc | Composições e métodos para administração de componentes terapêuticos |
US11981912B2 (en) | 2019-12-10 | 2024-05-14 | Takeda Pharma ceutical Company Limited | Adeno associated virus vectors for the treatment of hunter disease |
CA3174863A1 (en) * | 2020-04-14 | 2021-10-21 | Ana BUJ BELLO | Vectors for the treatment of acid ceramidase deficiency |
Family Cites Families (26)
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US5672344A (en) | 1987-12-30 | 1997-09-30 | The Regents Of The University Of Michigan | Viral-mediated gene transfer system |
US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
CA2166118C (en) | 1993-06-24 | 2007-04-17 | Frank L. Graham | Adenovirus vectors for gene therapy |
HU223733B1 (hu) | 1993-10-25 | 2004-12-28 | Canji, Inc. | Rekombináns adenovírus vektor és eljárás alkalmazására |
WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
ATE386131T1 (de) | 1994-04-13 | 2008-03-15 | Univ Rockefeller | Aav-vermittelte überbringung von dna in zellen des nervensystems |
US6274309B1 (en) | 1996-07-26 | 2001-08-14 | Richard Kolesnick | Method for the modulation of acid-sphingomyelinase-related apoptosis |
US20040076613A1 (en) * | 2000-11-03 | 2004-04-22 | Nicholas Mazarakis | Vector system |
US20050026832A1 (en) * | 1997-11-25 | 2005-02-03 | Genentech, Inc. | Fibroblast growth factor-19 (FGF-19) nucleic acids and polypeptides and methods of use for the treatment of obesity and related disorders |
US6544785B1 (en) | 1998-09-14 | 2003-04-08 | Mount Sinai School Of Medicine Of New York University | Helper-free rescue of recombinant negative strand RNA viruses |
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US20010031741A1 (en) * | 2000-02-17 | 2001-10-18 | Robin Ziegler | Methods for treatment of lysosomal storage diseases |
US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
WO2002024932A2 (en) | 2000-09-18 | 2002-03-28 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
US20040258666A1 (en) | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
CA2528427A1 (en) | 2003-06-12 | 2004-12-23 | Genzyme Corporation | Modified human acid sphingomyelinase having increased activity, and methods for making the same |
US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
US20060171926A1 (en) * | 2004-04-30 | 2006-08-03 | Passini Marco A | Gene therapy for neurometabolic disorders |
CN101212988A (zh) * | 2005-05-02 | 2008-07-02 | 建新公司 | 神经代谢疾病的基因治疗 |
JP5706602B2 (ja) * | 2006-02-08 | 2015-04-22 | ジェンザイム・コーポレーション | ニーマンピック病a型に対する遺伝子治療 |
DK1988823T3 (en) * | 2006-02-09 | 2018-12-03 | Genzyme Corp | SLOW INTRAVENTRICULAR ADMINISTRATION |
PL3252161T3 (pl) | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
KR20230164773A (ko) | 2009-08-28 | 2023-12-04 | 이칸 스쿨 오브 메디슨 엣 마운트 시나이 | 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법 |
-
2008
- 2008-06-02 PL PL17168189T patent/PL3252161T3/pl unknown
- 2008-06-02 EP EP08756596.6A patent/EP2158322B1/en active Active
- 2008-06-02 LT LTEP08756596.6T patent/LT2158322T/lt unknown
- 2008-06-02 PL PL08756596T patent/PL2158322T3/pl unknown
- 2008-06-02 DK DK08756596.6T patent/DK2158322T3/en active
- 2008-06-02 ES ES17168189T patent/ES2905616T3/es active Active
- 2008-06-02 WO PCT/US2008/065481 patent/WO2008154198A1/en active Application Filing
- 2008-06-02 EP EP17168189.3A patent/EP3252161B1/en active Active
- 2008-06-02 PT PT171681893T patent/PT3252161T/pt unknown
- 2008-06-02 PT PT87565966T patent/PT2158322T/pt unknown
- 2008-06-02 HU HUE08756596A patent/HUE035779T2/en unknown
- 2008-06-02 ES ES08756596.6T patent/ES2635726T3/es active Active
- 2008-06-02 SI SI200831847T patent/SI2158322T1/sl unknown
- 2008-06-06 AR ARP080102421A patent/AR070000A1/es unknown
-
2009
- 2009-12-04 US US12/631,336 patent/US8796236B2/en active Active
- 2009-12-06 IL IL202522A patent/IL202522A/en active IP Right Grant
-
2014
- 2014-07-10 US US14/328,621 patent/US11369693B2/en active Active
-
2017
- 2017-06-14 IL IL252898A patent/IL252898B/en active IP Right Grant
- 2017-07-31 HR HRP20171165TT patent/HRP20171165T1/hr unknown
- 2017-08-03 CY CY20171100836T patent/CY1119295T1/el unknown
-
2019
- 2019-05-19 IL IL266734A patent/IL266734B/en unknown
Also Published As
Publication number | Publication date |
---|---|
IL252898A0 (en) | 2017-08-31 |
DK2158322T3 (en) | 2017-08-28 |
IL266734B (en) | 2021-10-31 |
PT2158322T (pt) | 2017-08-09 |
PL2158322T3 (pl) | 2017-10-31 |
CY1119295T1 (el) | 2018-02-14 |
US11369693B2 (en) | 2022-06-28 |
EP3252161A1 (en) | 2017-12-06 |
IL266734A (en) | 2019-07-31 |
PL3252161T3 (pl) | 2022-03-07 |
US20100173979A1 (en) | 2010-07-08 |
EP2158322A4 (en) | 2011-06-01 |
US8796236B2 (en) | 2014-08-05 |
EP3252161B1 (en) | 2021-11-17 |
LT2158322T (lt) | 2017-08-25 |
IL202522A0 (en) | 2011-08-01 |
EP2158322A1 (en) | 2010-03-03 |
PT3252161T (pt) | 2022-02-01 |
HUE035779T2 (en) | 2018-05-28 |
EP2158322B1 (en) | 2017-05-03 |
ES2905616T3 (es) | 2022-04-11 |
ES2635726T3 (es) | 2017-10-04 |
AR070000A1 (es) | 2010-03-10 |
IL252898B (en) | 2019-06-30 |
US20150151007A1 (en) | 2015-06-04 |
WO2008154198A1 (en) | 2008-12-18 |
IL202522A (en) | 2017-10-31 |
SI2158322T1 (sl) | 2017-10-30 |
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