JP2017518043A5 - - Google Patents

Info

Publication number

JP2017518043A5

JP2017518043A5 JP2016564998A JP2016564998A JP2017518043A5 JP 2017518043 A5 JP2017518043 A5 JP 2017518043A5 JP 2016564998 A JP2016564998 A JP 2016564998A JP 2016564998 A JP2016564998 A JP 2016564998A JP 2017518043 A5 JP2017518043 A5 JP 2017518043A5

Authority

JP

Japan

Prior art keywords

complex

polynucleotide

antisense strand

seq

gene

Prior art date

2015-05-01

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.)

Granted

Links

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• 108091033319 polynucleotide Proteins 0.000 claims description 24
• 239000002157 polynucleotide Substances 0.000 claims description 24
• 102000040430 polynucleotide Human genes 0.000 claims description 24
• 230000000692 anti-sense effect Effects 0.000 claims description 14
• 239000008194 pharmaceutical composition Substances 0.000 claims description 10
• 229920001184 polypeptide Polymers 0.000 claims description 10
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 10
• 102000004196 processed proteins & peptides Human genes 0.000 claims description 10
• 235000001014 amino acid Nutrition 0.000 claims description 8
• 150000001413 amino acids Chemical class 0.000 claims description 8
• 210000003169 central nervous system Anatomy 0.000 claims description 8
• 230000000295 complement effect Effects 0.000 claims description 8
• 108020004999 messenger RNA Proteins 0.000 claims description 8
• 101150081618 nox gene Proteins 0.000 claims description 8
• 239000002773 nucleotide Substances 0.000 claims description 8
• 125000003729 nucleotide group Chemical group 0.000 claims description 8
• 108090000623 proteins and genes Proteins 0.000 claims description 7
• 108010082699 NADPH Oxidase 4 Proteins 0.000 claims description 6
• 102100021872 NADPH oxidase 4 Human genes 0.000 claims description 6
• 201000010099 disease Diseases 0.000 claims description 6
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 6
• 238000001990 intravenous administration Methods 0.000 claims description 6
• 238000000034 method Methods 0.000 claims description 5
• 102100025621 Cytochrome b-245 heavy chain Human genes 0.000 claims description 4
• 102000004019 NADPH Oxidase 1 Human genes 0.000 claims description 4
• 108090000424 NADPH Oxidase 1 Proteins 0.000 claims description 4
• 108010082739 NADPH Oxidase 2 Proteins 0.000 claims description 4
• 108091081021 Sense strand Proteins 0.000 claims description 4
• 125000003275 alpha amino acid group Chemical group 0.000 claims description 4
• 238000001802 infusion Methods 0.000 claims description 3
• 238000010254 subcutaneous injection Methods 0.000 claims description 3
• 239000007929 subcutaneous injection Substances 0.000 claims description 3
• 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 2
• 108091034117 Oligonucleotide Proteins 0.000 claims description 2
• 208000006011 Stroke Diseases 0.000 claims description 2
• 238000007792 addition Methods 0.000 claims description 2
• 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims description 2
• 238000012217 deletion Methods 0.000 claims description 2
• 230000037430 deletion Effects 0.000 claims description 2
• 239000003937 drug carrier Substances 0.000 claims description 2
• 208000014674 injury Diseases 0.000 claims description 2
• 238000003780 insertion Methods 0.000 claims description 2
• 230000037431 insertion Effects 0.000 claims description 2
• 208000028867 ischemia Diseases 0.000 claims description 2
• 208000004296 neuralgia Diseases 0.000 claims description 2
• 230000004770 neurodegeneration Effects 0.000 claims description 2
• 208000015122 neurodegenerative disease Diseases 0.000 claims description 2
• 208000021722 neuropathic pain Diseases 0.000 claims description 2
• 208000020016 psychiatric disease Diseases 0.000 claims description 2
• 230000001698 pyrogenic effect Effects 0.000 claims description 2
• 238000006467 substitution reaction Methods 0.000 claims description 2
• 230000008733 trauma Effects 0.000 claims description 2
• 239000000203 mixture Substances 0.000 claims 3
• 239000002131 composite material Substances 0.000 claims 2
• 210000004899 c-terminal region Anatomy 0.000 claims 1
• 108091033409 CRISPR Proteins 0.000 description 1
• 238000010354 CRISPR gene editing Methods 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