JP2015518711A5 - - Google Patents

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Publication number: JP2015518711A5
Authority: JP; Japan
Prior art keywords: oligonucleotide; nucleotides; nucleotide; 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

Application number

JP2015512845A

Other languages

English (en)

Japanese (ja)

Other versions

JP2015518711A (ja

Filing date

2013-05-16

Publication date

2016-06-30

2013-05-16 Application filed filed Critical

2013-05-16 Priority claimed from PCT/US2013/041385 external-priority patent/WO2013173601A1/en

2015-07-06 Publication of JP2015518711A publication Critical patent/JP2015518711A/ja

2016-06-30 Publication of JP2015518711A5 publication Critical patent/JP2015518711A5/ja

Status Pending legal-status Critical Current

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108091034117 Oligonucleotide Proteins 0.000 claims description 103
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 23
239000005547 deoxyribonucleotide Substances 0.000 claims description 18
125000002637 deoxyribonucleotide group Chemical group 0.000 claims description 16
101150035467 BDNF gene Proteins 0.000 claims description 14
101100465401 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) SCL1 gene Proteins 0.000 claims description 11
230000000295 complement effect Effects 0.000 claims description 10
206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims description 9
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
108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims description 6
208000018737 Parkinson disease Diseases 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
208000024827 Alzheimer disease Diseases 0.000 claims description 5
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
230000004770 neurodegeneration Effects 0.000 claims description 3
108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 2
108091028664 Ribonucleotide Proteins 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
208000015122 neurodegenerative disease Diseases 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
125000002652 ribonucleotide group Chemical group 0.000 claims description 2
238000011144 upstream manufacturing Methods 0.000 claims description 2
238000004132 cross linking Methods 0.000 claims 2
108091092195 Intron Proteins 0.000 claims 1
230000003247 decreasing effect Effects 0.000 claims 1
150000003431 steroids Chemical class 0.000 claims 1
238000000034 method Methods 0.000 description 7
102000004219 Brain-derived neurotrophic factor Human genes 0.000 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 description 4
208000023105 Huntington disease Diseases 0.000 description 2
101000739876 Homo sapiens Brain-derived neurotrophic factor Proteins 0.000 description 1
239000007853 buffer solution Substances 0.000 description 1
201000010099 disease Diseases 0.000 description 1
208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1
102000051542 human BDNF Human genes 0.000 description 1
229940077456 human brain-derived neurotrophic factor Drugs 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

JP2015512845A 2012-05-16 2013-05-16 Ｂｄｎｆ発現を調節するための組成物及び方法 Pending JP2015518711A (ja)

Applications Claiming Priority (3)

Application Number	Priority Date	Filing Date	Title
US201261648058P	2012-05-16	2012-05-16
US61/648,058		2012-05-16
PCT/US2013/041385 WO2013173601A1 (en)	2012-05-16	2013-05-16	Compositions and methods for modulating bdnf expression

Publications (2)

Publication Number	Publication Date
JP2015518711A JP2015518711A (ja)	2015-07-06
JP2015518711A5 true JP2015518711A5 (2)	2016-06-30

Family

ID=49584294

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
JP2015512845A Pending JP2015518711A (ja)	2012-05-16	2013-05-16	Ｂｄｎｆ発現を調節するための組成物及び方法

Country Status (9)

Country	Link
US (1)	US20150133529A1 (2)
EP (1)	EP2849800A4 (2)
JP (1)	JP2015518711A (2)
CN (1)	CN104602714A (2)
AU (1)	AU2013262702A1 (2)
BR (1)	BR112014028647A2 (2)
CA (1)	CA2873772A1 (2)
EA (1)	EA201492117A1 (2)
WO (1)	WO2013173601A1 (2)

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US9920317B2 (en)	2010-11-12	2018-03-20	The General Hospital Corporation	Polycomb-associated non-coding RNAs
EP3260540A1 (en)	2010-11-12	2017-12-27	The General Hospital Corporation	Polycomb-associated non-coding rnas
CA2873769A1 (en)	2012-05-16	2013-11-21	Rana Therapeutics Inc.	Compositions and methods for modulating hemoglobin gene family expression
US10837014B2 (en)	2012-05-16	2020-11-17	Translate Bio Ma, Inc.	Compositions and methods for modulating SMN gene family expression
CA2873766A1 (en)	2012-05-16	2013-11-21	Rana Therapeutics Inc.	Compositions and methods for modulating atp2a2 expression
AU2013262709A1 (en)	2012-05-16	2015-01-22	Rana Therapeutics, Inc.	Compositions and methods for modulating MECP2 expression
JP2015523854A (ja)	2012-05-16	2015-08-20	ラナセラピューティクスインコーポレイテッド	Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法
BR112014028634A2 (pt)	2012-05-16	2017-06-27	Rana Therapeutics Inc	composições e métodos para modulação da expressão de utrn
AU2014274730A1 (en) *	2013-06-07	2016-01-21	Rana Therapeutics, Inc.	Compositions and methods for modulating FOXP3 expression
JP2016531570A (ja)	2013-08-16	2016-10-13	ラナセラピューティクスインコーポレイテッド	ユークロマチン領域を標的とするオリゴヌクレオチド
JP6618910B2 (ja) *	2013-09-05	2019-12-11	サレプタセラピューティクス，インコーポレイテッド	酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含
CN108064175A (zh) *	2014-08-04	2018-05-22	米拉根医疗股份有限公司	Myh7b的抑制剂及其用途
CA2966044A1 (en)	2014-10-30	2016-05-06	The General Hospital Corporation	Methods for modulating atrx-dependent gene repression
WO2016149455A2 (en)	2015-03-17	2016-09-22	The General Hospital Corporation	The rna interactome of polycomb repressive complex 1 (prc1)
AU2017213404A1 (en)	2016-01-29	2018-09-20	Kyowa Kirin Co., Ltd.	Nucleic acid conjugate
CA3021267A1 (en)	2016-04-18	2017-10-26	Sarepta Therapeutics, Inc.	Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
TWI746590B (zh)	2016-06-30	2021-11-21	日商協和麒麟股份有限公司	核酸複合體
US11584932B2 (en)	2016-11-01	2023-02-21	The Research Foundation For The State University Of New York	5-halouracil-modified microRNAs and their use in the treatment of cancer
EP4035659A1 (en)	2016-11-29	2022-08-03	PureTech LYT, Inc.	Exosomes for delivery of therapeutic agents
CN108315350B (zh) *	2018-03-01	2021-08-27	昆明医科大学	过表达cox5a/低表达bdnf转基因鼠模型及其构建方法与应用
WO2021262919A2 (en)	2020-06-26	2021-12-30	The Research Foundation For The State University Of New York	5-halouracil-modified micrornas and their use in the treatment of cancer
US20230287427A1 (en) *	2020-07-29	2023-09-14	The General Hospital Corporation	Inhibition of lncExACT1 to Treat Heart Disease
EP4353825A1 (en) *	2022-10-10	2024-04-17	bisy GmbH	Modified promoter sequences

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Publication number	Priority date	Publication date	Assignee	Title
EP0593663A4 (en) *	1991-07-03	1996-10-30	Regeneron Pharma	Method and assay system for neurotrophin activity
US6794499B2 (en) *	1997-09-12	2004-09-21	Exiqon A/S	Oligonucleotide analogues
US7715989B2 (en) *	1998-04-03	2010-05-11	Elitech Holding B.V.	Systems and methods for predicting oligonucleotide melting temperature (TmS)
US6825338B2 (en) *	2001-03-30	2004-11-30	Isis Pharmaceuticals, Inc.	Labeled oligonucleotides, methods for making same, and compounds useful therefor
US20050176025A1 (en) *	2001-05-18	2005-08-11	Sirna Therapeutics, Inc.	RNA interference mediated inhibition of B-cell CLL/Lymphoma-2 (BCL-2) gene expression using short interfering nucleic acid (siNA)
WO2003085110A2 (en) *	2002-04-05	2003-10-16	Santaris Pharma A/S	Oligomeric compounds for the modulation hif-1alpha expression
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US20080176793A1 (en) *	2006-09-18	2008-07-24	The Regents Of The University Of California	Upregulating activity or expression of bdnf to mitigate cognitive impairment in asymptomatic huntington's subjects
WO2008060375A2 (en) *	2006-10-06	2008-05-22	The Regents Of The University Of Californina	Upregulating bdnf levels to mitigate mental retardation
WO2010093904A2 (en) *	2009-02-12	2010-08-19	Curna, Inc.	Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
KR101138048B1 (ko) *	2009-11-06	2012-04-23	성균관대학교산학협력단	Ｂｄｎｆ의 발현을 증가시키는 신규 펩타이드 및 이를 포함하는 알츠하이머병 또는 파킨슨병의 예방 및 치료용 약학 조성물
KR101235256B1 (ko) *	2010-09-13	2013-02-21	서울대학교산학협력단	ｍｉＲＮＡ를 타겟으로 한 신경퇴행성 질환 치료
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2013
- 2013-05-16 EA EA201492117A patent/EA201492117A1/ru unknown
- 2013-05-16 AU AU2013262702A patent/AU2013262702A1/en not_active Abandoned
- 2013-05-16 EP EP13790378.7A patent/EP2849800A4/en not_active Withdrawn
- 2013-05-16 CN CN201380037592.8A patent/CN104602714A/zh active Pending
- 2013-05-16 JP JP2015512845A patent/JP2015518711A/ja active Pending
- 2013-05-16 CA CA2873772A patent/CA2873772A1/en not_active Abandoned
- 2013-05-16 WO PCT/US2013/041385 patent/WO2013173601A1/en not_active Ceased
- 2013-05-16 BR BR112014028647A patent/BR112014028647A2/pt not_active IP Right Cessation
- 2013-05-16 US US14/401,234 patent/US20150133529A1/en not_active Abandoned

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