JP2020505032A5 - - Google Patents

Info

Publication number

JP2020505032A5

JP2020505032A5 JP2019538617A JP2019538617A JP2020505032A5 JP 2020505032 A5 JP2020505032 A5 JP 2020505032A5 JP 2019538617 A JP2019538617 A JP 2019538617A JP 2019538617 A JP2019538617 A JP 2019538617A JP 2020505032 A5 JP2020505032 A5 JP 2020505032A5

Authority

JP

Japan

Prior art keywords

target molecule

molecule

target

sirna

effector

Prior art date

2018-01-18

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

• Espacenet
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• 108020004459 Small interfering RNA Proteins 0.000 claims description 26
• 239000012636 effector Substances 0.000 claims description 22
• 150000007523 nucleic acids Chemical class 0.000 claims description 18
• 108020004707 nucleic acids Proteins 0.000 claims description 17
• 102000039446 nucleic acids Human genes 0.000 claims description 17
• 239000003446 ligand Substances 0.000 claims description 15
• 108091034117 Oligonucleotide Proteins 0.000 claims description 14
• 230000000692 anti-sense effect Effects 0.000 claims description 11
• 108091081021 Sense strand Proteins 0.000 claims description 10
• 230000014509 gene expression Effects 0.000 claims description 8
• 238000000338 in vitro Methods 0.000 claims description 6
• 230000003278 mimic effect Effects 0.000 claims description 6
• 108091027967 Small hairpin RNA Proteins 0.000 claims description 5
• 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 claims description 5
• 230000004048 modification Effects 0.000 claims description 5
• 238000012986 modification Methods 0.000 claims description 5
• 239000004055 small Interfering RNA Substances 0.000 claims description 5
• 230000003834 intracellular effect Effects 0.000 claims description 4
• 239000002679 microRNA Substances 0.000 claims description 4
• 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 3
• 108090000994 Catalytic RNA Proteins 0.000 claims description 3
• 102000053642 Catalytic RNA Human genes 0.000 claims description 3
• 108700011259 MicroRNAs Proteins 0.000 claims description 3
• 239000012190 activator Substances 0.000 claims description 3
• 239000000074 antisense oligonucleotide Substances 0.000 claims description 3
• 238000012230 antisense oligonucleotides Methods 0.000 claims description 3
• 210000004369 blood Anatomy 0.000 claims description 3
• 239000008280 blood Substances 0.000 claims description 3
• 235000014633 carbohydrates Nutrition 0.000 claims description 3
• 230000003308 immunostimulating effect Effects 0.000 claims description 3
• 108091092562 ribozyme Proteins 0.000 claims description 3
• 210000002966 serum Anatomy 0.000 claims description 3
• 239000002924 silencing RNA Substances 0.000 claims description 3
• 239000002777 nucleoside Substances 0.000 claims description 2
• 150000003833 nucleoside derivatives Chemical class 0.000 claims description 2
• 125000003835 nucleoside group Chemical group 0.000 claims description 2
• 235000000346 sugar Nutrition 0.000 claims description 2
• 150000008163 sugars Chemical class 0.000 claims description 2
• 108091033409 CRISPR Proteins 0.000 claims 1
• 238000010354 CRISPR gene editing Methods 0.000 claims 1
• 0 CC(C)CC*C(*1*)OC(CO)C(*)C1O*(C(*)C1*C2OC(CO)C(*)C(*)C2*)OC(C*)C1O Chemical compound CC(C)CC*C(*1*)OC(CO)C(*)C1O*(C(*)C1*C2OC(CO)C(*)C(*)C2*)OC(C*)C1O 0.000 description 6
• 210000001163 endosome Anatomy 0.000 description 2
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• 108020000948 Antisense Oligonucleotides Proteins 0.000 description 1
• 108091028043 Nucleic acid sequence Proteins 0.000 description 1
• 108091005804 Peptidases Proteins 0.000 description 1
• 239000004365 Protease Substances 0.000 description 1
• 102100037486 Reverse transcriptase/ribonuclease H Human genes 0.000 description 1
• -1 U1 adapters Substances 0.000 description 1
• 238000007792 addition Methods 0.000 description 1
• 239000000556 agonist Substances 0.000 description 1
• 210000004027 cell Anatomy 0.000 description 1
• 229940002612 prodrug Drugs 0.000 description 1
• 239000000651 prodrug Substances 0.000 description 1
• 108090000623 proteins and genes Proteins 0.000 description 1