JP2006515864A5 - - Google Patents
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- JP2006515864A5 JP2006515864A5 JP2005510322A JP2005510322A JP2006515864A5 JP 2006515864 A5 JP2006515864 A5 JP 2006515864A5 JP 2005510322 A JP2005510322 A JP 2005510322A JP 2005510322 A JP2005510322 A JP 2005510322A JP 2006515864 A5 JP2006515864 A5 JP 2006515864A5
- Authority
- JP
- Japan
- Prior art keywords
- small interfering
- interfering rna
- medical system
- neurodegenerative disease
- complementary
- 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.)
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- 108020004459 Small interfering RNA Proteins 0.000 claims description 61
- 239000004055 small Interfering RNA Substances 0.000 claims description 54
- 108020004999 messenger RNA Proteins 0.000 claims description 27
- 230000000295 complement effect Effects 0.000 claims description 23
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 9
- 108010032963 Ataxin-1 Proteins 0.000 claims description 7
- 102000007372 Ataxin-1 Human genes 0.000 claims description 4
- 238000000034 method Methods 0.000 claims 44
- 230000004770 neurodegeneration Effects 0.000 claims 32
- 208000015122 neurodegenerative disease Diseases 0.000 claims 32
- 238000007917 intracranial administration Methods 0.000 claims 15
- 108090000623 proteins and genes Proteins 0.000 claims 13
- 102000004169 proteins and genes Human genes 0.000 claims 10
- 108010032951 Ataxin2 Proteins 0.000 claims 9
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 7
- 210000004556 brain Anatomy 0.000 claims 7
- 210000003591 cerebellar nuclei Anatomy 0.000 claims 7
- 108010032947 Ataxin-3 Proteins 0.000 claims 6
- 102000007370 Ataxin2 Human genes 0.000 claims 6
- 201000008163 Dentatorubral pallidoluysian atrophy Diseases 0.000 claims 6
- 108050004784 Huntingtin Proteins 0.000 claims 6
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 claims 6
- 238000001802 infusion Methods 0.000 claims 6
- 102000007371 Ataxin-3 Human genes 0.000 claims 4
- 208000002569 Machado-Joseph Disease Diseases 0.000 claims 4
- 208000036834 Spinocerebellar ataxia type 3 Diseases 0.000 claims 4
- 210000003710 cerebral cortex Anatomy 0.000 claims 4
- 208000024827 Alzheimer disease Diseases 0.000 claims 3
- 102000004321 Atrophin-1 Human genes 0.000 claims 3
- 108090000806 Atrophin-1 Proteins 0.000 claims 3
- 102100021257 Beta-secretase 1 Human genes 0.000 claims 3
- 108020004414 DNA Proteins 0.000 claims 3
- 102000004190 Enzymes Human genes 0.000 claims 3
- 108090000790 Enzymes Proteins 0.000 claims 3
- 101000894895 Homo sapiens Beta-secretase 1 Proteins 0.000 claims 3
- 102000016252 Huntingtin Human genes 0.000 claims 3
- 208000023105 Huntington disease Diseases 0.000 claims 3
- 208000018737 Parkinson disease Diseases 0.000 claims 3
- 201000003622 Spinocerebellar ataxia type 2 Diseases 0.000 claims 3
- 102000003802 alpha-Synuclein Human genes 0.000 claims 3
- 108090000185 alpha-Synuclein Proteins 0.000 claims 3
- 239000013612 plasmid Substances 0.000 claims 3
- 210000000463 red nucleus Anatomy 0.000 claims 3
- 201000003624 spinocerebellar ataxia type 1 Diseases 0.000 claims 3
- 102000013455 Amyloid beta-Peptides Human genes 0.000 claims 2
- 108010090849 Amyloid beta-Peptides Proteins 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 210000004227 basal ganglia Anatomy 0.000 claims 2
- 210000001159 caudate nucleus Anatomy 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 210000001577 neostriatum Anatomy 0.000 claims 2
- 230000003204 osmotic effect Effects 0.000 claims 2
- 230000001936 parietal effect Effects 0.000 claims 2
- 210000002637 putamen Anatomy 0.000 claims 2
- 210000004281 subthalamic nucleus Anatomy 0.000 claims 2
- 241000282412 Homo Species 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 241001465754 Metazoa Species 0.000 claims 1
- 241000700584 Simplexvirus Species 0.000 claims 1
- 210000001638 cerebellum Anatomy 0.000 claims 1
- 230000002490 cerebral effect Effects 0.000 claims 1
- 150000001875 compounds Chemical class 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000013507 mapping Methods 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
- 210000003523 substantia nigra Anatomy 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 101000873082 Homo sapiens Ataxin-1 Proteins 0.000 description 5
- 102000056417 human ATXN1 Human genes 0.000 description 5
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 4
- 230000008685 targeting Effects 0.000 description 4
- 230000035897 transcription Effects 0.000 description 3
- 238000013518 transcription Methods 0.000 description 3
- 108091034117 Oligonucleotide Proteins 0.000 description 2
- 239000012634 fragment Substances 0.000 description 2
- 238000000338 in vitro Methods 0.000 description 2
- 239000002773 nucleotide Substances 0.000 description 2
- 125000003729 nucleotide group Chemical group 0.000 description 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 1
- 102000016911 Deoxyribonucleases Human genes 0.000 description 1
- 108010053770 Deoxyribonucleases Proteins 0.000 description 1
- 230000007022 RNA scission Effects 0.000 description 1
- 102000006382 Ribonucleases Human genes 0.000 description 1
- 108010083644 Ribonucleases Proteins 0.000 description 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 description 1
- 230000000692 anti-sense effect Effects 0.000 description 1
- 238000006243 chemical reaction Methods 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- 238000002515 oligonucleotide synthesis Methods 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US42938702P | 2002-11-26 | 2002-11-26 | |
| US60/429,387 | 2002-11-26 | ||
| US44461403P | 2003-02-03 | 2003-02-03 | |
| US60/444,614 | 2003-02-03 | ||
| PCT/US2003/037650 WO2004047872A2 (en) | 2002-11-26 | 2003-11-26 | Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna) |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011162422A Division JP2012006938A (ja) | 2002-11-26 | 2011-07-25 | ｓｉＲＮＡの頭蓋内送達を通した神経変性疾患の治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2006515864A JP2006515864A (ja) | 2006-06-08 |
| JP2006515864A5 true JP2006515864A5 (https=) | 2010-09-16 |
| JP4868854B2 JP4868854B2 (ja) | 2012-02-01 |
Family
ID=32397198
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2005510322A Expired - Fee Related JP4868854B2 (ja) | 2002-11-26 | 2003-11-26 | ｓｉＲＮＡの頭蓋内送達を通した神経変性疾患の治療 |
| JP2011162422A Pending JP2012006938A (ja) | 2002-11-26 | 2011-07-25 | ｓｉＲＮＡの頭蓋内送達を通した神経変性疾患の治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011162422A Pending JP2012006938A (ja) | 2002-11-26 | 2011-07-25 | ｓｉＲＮＡの頭蓋内送達を通した神経変性疾患の治療 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US7605249B2 (https=) |
| EP (4) | EP1832292A1 (https=) |
| JP (2) | JP4868854B2 (https=) |
| AT (1) | ATE550025T1 (https=) |
| AU (1) | AU2003293035A1 (https=) |
| CA (1) | CA2507606A1 (https=) |
| WO (1) | WO2004047872A2 (https=) |
Families Citing this family (97)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2002326589B2 (en) * | 2001-08-07 | 2008-06-05 | University Of Delaware | Compositions and methods for the prevention and treatment of Huntington's disease |
| US20040096880A1 (en) * | 2001-08-07 | 2004-05-20 | Kmiec Eric B. | Compositions and methods for the treatment of diseases exhibiting protein misassembly and aggregation |
| US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US20040241854A1 (en) | 2002-08-05 | 2004-12-02 | Davidson Beverly L. | siRNA-mediated gene silencing |
| US20050255086A1 (en) * | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
| US20050106731A1 (en) * | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US7618948B2 (en) | 2002-11-26 | 2009-11-17 | Medtronic, Inc. | Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA |
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| US7829694B2 (en) * | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| AU2003301030A1 (en) * | 2002-12-17 | 2004-07-22 | Victor Miller | Sirna-mediated gene silencing |
| US7732591B2 (en) * | 2003-11-25 | 2010-06-08 | Medtronic, Inc. | Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna |
| US7994149B2 (en) | 2003-02-03 | 2011-08-09 | Medtronic, Inc. | Method for treatment of Huntington's disease through intracranial delivery of sirna |
| WO2004101787A1 (ja) * | 2003-05-14 | 2004-11-25 | Japan Science And Technology Agency | ハンチンチン遺伝子の発現抑制 |
| US7595306B2 (en) * | 2003-06-09 | 2009-09-29 | Alnylam Pharmaceuticals Inc | Method of treating neurodegenerative disease |
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| CN200970210Y (zh) | 2006-10-12 | 2007-11-07 | 王会才 | 拖把清洗脱水机 |
| WO2008143774A2 (en) | 2007-05-01 | 2008-11-27 | University Of Massachusetts | Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy |
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2003
- 2003-11-25 US US10/721,693 patent/US7605249B2/en not_active Expired - Fee Related
- 2003-11-26 JP JP2005510322A patent/JP4868854B2/ja not_active Expired - Fee Related
- 2003-11-26 CA CA002507606A patent/CA2507606A1/en not_active Abandoned
- 2003-11-26 EP EP07003774A patent/EP1832292A1/en not_active Withdrawn
- 2003-11-26 EP EP09013626A patent/EP2145628B1/en not_active Expired - Lifetime
- 2003-11-26 AT AT09013626T patent/ATE550025T1/de active
- 2003-11-26 EP EP03790026A patent/EP1569662A2/en not_active Withdrawn
- 2003-11-26 EP EP07003775A patent/EP1829547A3/en not_active Withdrawn
- 2003-11-26 AU AU2003293035A patent/AU2003293035A1/en not_active Abandoned
- 2003-11-26 WO PCT/US2003/037650 patent/WO2004047872A2/en not_active Ceased
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2009
- 2009-08-27 US US12/549,110 patent/US8119611B2/en not_active Expired - Fee Related
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2011
- 2011-07-25 JP JP2011162422A patent/JP2012006938A/ja active Pending
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