JP2015518712A5 - - Google Patents

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Publication number

JP2015518712A5

JP2015518712A5 JP2015512847A JP2015512847A JP2015518712A5 JP 2015518712 A5 JP2015518712 A5 JP 2015518712A5 JP 2015512847 A JP2015512847 A JP 2015512847A JP 2015512847 A JP2015512847 A JP 2015512847A JP 2015518712 A5 JP2015518712 A5 JP 2015518712A5

Authority

JP

Japan

Prior art keywords

oligonucleotide

nucleotide

nucleotides

sequence

composition

Prior art date

2013-05-16

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.)

Pending

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• 125000003729 nucleotide group Chemical group 0.000 claims description 78
• 239000002773 nucleotide Substances 0.000 claims description 69
• 239000000203 mixture Substances 0.000 claims description 22
• 101150083522 MECP2 gene Proteins 0.000 claims description 21
• 239000005547 deoxyribonucleotide Substances 0.000 claims description 18
• 125000002637 deoxyribonucleotide group Chemical group 0.000 claims description 16
• 101100465401 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) SCL1 gene Proteins 0.000 claims description 11
• 230000000295 complement effect Effects 0.000 claims description 10
• NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical class C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims description 8
• 230000014509 gene expression Effects 0.000 claims description 7
• 102100039124 Methyl-CpG-binding protein 2 Human genes 0.000 claims description 6
• 108090000623 proteins and genes Proteins 0.000 claims description 6
• RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 6
• YBJHBAHKTGYVGT-ZKWXMUAHSA-N (+)-Biotin Chemical group N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 claims description 4
• 108700011259 MicroRNAs Proteins 0.000 claims description 4
• OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 4
• 239000002679 microRNA Substances 0.000 claims description 4
• 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 2
• 208000009575 Angelman syndrome Diseases 0.000 claims description 2
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• 208000006289 Rett Syndrome Diseases 0.000 claims description 2
• 108091028664 Ribonucleotide Proteins 0.000 claims description 2
• 201000001875 X-linked intellectual disability-psychosis-macroorchidism syndrome Diseases 0.000 claims description 2
• 229960002685 biotin Drugs 0.000 claims description 2
• 235000020958 biotin Nutrition 0.000 claims description 2
• 239000011616 biotin Substances 0.000 claims description 2
• 229940104302 cytosine Drugs 0.000 claims description 2
• 239000003937 drug carrier Substances 0.000 claims description 2
• 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 2
• 239000008194 pharmaceutical composition Substances 0.000 claims description 2
• 108090000765 processed proteins & peptides Proteins 0.000 claims description 2
• 239000002336 ribonucleotide Substances 0.000 claims description 2
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• 108091092195 Intron Proteins 0.000 claims 1
• 150000003431 steroids Chemical class 0.000 claims 1
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• 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 4
• 101001033726 Homo sapiens Methyl-CpG-binding protein 2 Proteins 0.000 description 1
• 239000007853 buffer solution Substances 0.000 description 1
• 102000049101 human MECP2 Human genes 0.000 description 1
• 230000001404 mediated effect Effects 0.000 description 1
• 125000002345 steroid group Chemical group 0.000 description 1
• 230000001629 suppression Effects 0.000 description 1
• 230000003827 upregulation Effects 0.000 description 1