JP2016526529A5 - - Google Patents

Download PDF

Info

Publication number
JP2016526529A5
JP2016526529A5 JP2015560881A JP2015560881A JP2016526529A5 JP 2016526529 A5 JP2016526529 A5 JP 2016526529A5 JP 2015560881 A JP2015560881 A JP 2015560881A JP 2015560881 A JP2015560881 A JP 2015560881A JP 2016526529 A5 JP2016526529 A5 JP 2016526529A5
Authority
JP
Japan
Prior art keywords
nucleotides
strand
independently selected
nucleotide analogs
natural
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.)
Granted
Application number
JP2015560881A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016526529A (ja
JP6604544B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/JP2014/003208 external-priority patent/WO2014203518A1/en
Publication of JP2016526529A publication Critical patent/JP2016526529A/ja
Publication of JP2016526529A5 publication Critical patent/JP2016526529A5/ja
Application granted granted Critical
Publication of JP6604544B2 publication Critical patent/JP6604544B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2015560881A 2013-06-16 2014-06-16 エクソンスキッピング効果を有する二本鎖アンチセンス核酸 Active JP6604544B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361835634P 2013-06-16 2013-06-16
US61/835,634 2013-06-16
US201361836672P 2013-06-19 2013-06-19
US61/836,672 2013-06-19
PCT/JP2014/003208 WO2014203518A1 (en) 2013-06-16 2014-06-16 Double-stranded antisense nucleic acid with exon-skipping effect

Publications (3)

Publication Number Publication Date
JP2016526529A JP2016526529A (ja) 2016-09-05
JP2016526529A5 true JP2016526529A5 (enExample) 2017-08-17
JP6604544B2 JP6604544B2 (ja) 2019-11-13

Family

ID=52104269

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015560881A Active JP6604544B2 (ja) 2013-06-16 2014-06-16 エクソンスキッピング効果を有する二本鎖アンチセンス核酸

Country Status (7)

Country Link
US (1) US10190117B2 (enExample)
EP (1) EP3010514B1 (enExample)
JP (1) JP6604544B2 (enExample)
CN (1) CN105324119A (enExample)
AU (1) AU2014282666A1 (enExample)
CA (1) CA2915443A1 (enExample)
WO (1) WO2014203518A1 (enExample)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
US10428019B2 (en) 2010-09-24 2019-10-01 Wave Life Sciences Ltd. Chiral auxiliaries
EP3248982A1 (en) 2011-07-19 2017-11-29 Wave Life Sciences Ltd. Thiosulfonate reagents for the synthesis of functionalized nucleic acids
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
JP2015518712A (ja) 2012-05-16 2015-07-06 ラナ セラピューティクス インコーポレイテッド Mecp2発現を調節するための組成物及び方法
WO2013173638A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating smn gene family expression
CA2879023C (en) 2012-07-13 2017-03-28 Wave Life Sciences Japan Asymmetric auxiliary group
US10174328B2 (en) 2013-10-04 2019-01-08 Translate Bio Ma, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
DK3234134T3 (da) 2014-12-17 2020-07-27 Proqr Therapeutics Ii Bv Målrettet rna-redigering
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
US11116843B2 (en) 2015-09-25 2021-09-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
JP7749201B6 (ja) 2016-01-31 2025-10-21 ユニバーシティー オブ マサチューセッツ 分岐オリゴヌクレオチド
KR102608220B1 (ko) * 2016-05-06 2023-11-29 아이오니스 파마수티컬즈, 인코포레이티드 Glp-1 수용체 리간드 모이어티 컨쥬게이트된 올리고뉴클레오티드 및 이의 용도
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
KR20230175312A (ko) * 2016-07-05 2023-12-29 바이오마린 테크놀로지스 비.브이. 유전적 장애 치료를 위한, 특징이 개선된, 비사이클릭 스캐폴드 모이어티를 포함하는 프리-mrna 스플라이스 스위칭 또는 조정 올리고뉴클레오티드
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
ES2837076T3 (es) 2016-09-01 2021-06-29 Proqr Therapeutics Ii Bv Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente
JP7043078B2 (ja) 2016-09-23 2022-03-29 国立大学法人 東京医科歯科大学 血液脳関門通過型ヘテロ2本鎖核酸
JP7129702B2 (ja) 2016-09-29 2022-09-02 国立大学法人 東京医科歯科大学 オーバーハングを有する二本鎖核酸複合体
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
CN110799647A (zh) 2017-06-23 2020-02-14 马萨诸塞大学 双尾自递送sirna及相关的方法
US20200115710A1 (en) 2017-06-30 2020-04-16 National University Corporation Tokyo Medical And Dental University HETERO DOUBLE-STRANDED antimiR
CN119876131A (zh) * 2017-11-08 2025-04-25 Ionis制药公司 Glp-1受体配体部分缀合的寡核苷酸及其用途
US11674141B2 (en) 2018-02-28 2023-06-13 National University Corporation Tokyo Medical And Dental University Ischemic-lesion-site-specific gene therapy
EP3766972B1 (en) 2018-03-14 2025-04-23 Institute of Science Tokyo Nucleic acid complex
WO2019181946A1 (ja) 2018-03-19 2019-09-26 国立大学法人東京医科歯科大学 毒性が軽減した核酸
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
EP3833763A4 (en) 2018-08-10 2023-07-19 University of Massachusetts MODIFIED OLIGONUCLEOTIDES FOR TARGETING SNPS
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
JP2022523467A (ja) * 2019-01-18 2022-04-25 ユニバーシティ・オブ・マサチューセッツ 動的な薬物動態を修飾するアンカー
CN113677374A (zh) * 2019-04-08 2021-11-19 国立大学法人东京医科齿科大学 肌疾病治疗用药物组合物
CN114502730A (zh) 2019-08-09 2022-05-13 马萨诸塞大学 经化学修饰的靶向snp的寡核苷酸
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021070959A1 (ja) * 2019-10-11 2021-04-15 国立大学法人東京医科歯科大学 修飾ヘテロ核酸
US20230061751A1 (en) * 2020-01-17 2023-03-02 University Of Massachusetts Universal dynamic pharmacokinetic-modifying anchors
EP4123023A4 (en) * 2020-03-16 2024-07-17 National University Corporation Tokyo Medical and Dental University Heteronucleic acid containing morpholino nucleic acid
WO2022106695A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
EP4359539A4 (en) 2021-06-23 2025-05-14 University Of Massachusetts ANTI-FLT1 OLIGONUCLEOTIDE COMPOUNDS OPTIMIZED FOR THE TREATMENT OF PREECLAMPSIA AND OTHER ANGIOGENIC DISORDERS
JPWO2023022229A1 (enExample) * 2021-08-19 2023-02-23
WO2023105898A1 (ja) * 2021-12-07 2023-06-15 リードファーマ株式会社 アンチセンスオリゴヌクレオチド複合体
CN115806984B (zh) * 2022-10-18 2023-10-10 昆明理工大学 环状rna及载体和载体的应用
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH09110894A (ja) * 1995-10-17 1997-04-28 Soyaku Gijutsu Kenkyusho:Kk ハイブリッドdna/rnaオリゴヌクレオチド及び抗ウイルス剤
JP3781879B2 (ja) 1996-11-18 2006-05-31 武 今西 新規ヌクレオチド類縁体
US6043060A (en) 1996-11-18 2000-03-28 Imanishi; Takeshi Nucleotide analogues
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
KR100414936B1 (ko) 1997-09-12 2004-01-13 엑시콘 에이/에스 이환 및 삼환 뉴클레오시드, 뉴클레오타이드 및올리고뉴클레오타이드 동족체
US20010024808A1 (en) * 1998-09-10 2001-09-27 Millennium Pharmaceuticals, Inc., A Delaware Corporation Leptin induced genes
AU2001233176A1 (en) * 2000-01-31 2001-08-07 Shengfang Jin Resistance sequences and uses thereof
US20010039331A1 (en) * 2000-03-31 2001-11-08 Hunter John J. 16836, a novel human phospholipase C family member and uses thereof
CA2504694C (en) * 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP1569695B1 (en) 2002-11-13 2013-05-15 Genzyme Corporation Antisense modulation of apolipoprotein b expression
JP4731324B2 (ja) 2003-08-28 2011-07-20 武 今西 N−o結合性架橋構造型新規人工核酸
WO2005113571A2 (en) 2004-05-13 2005-12-01 The University Of North Carolina At Chapel Hill Methods for the delivery of oligomeric compounds
WO2006059507A1 (ja) 2004-11-30 2006-06-08 Sankyo Company, Limited 11β-HSD1アンチセンス化合物
WO2007107162A2 (en) * 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna
WO2007136989A2 (en) 2006-05-05 2007-11-29 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of dgat2
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
MX2009003729A (es) 2006-10-09 2009-04-22 Santaris Pharma As Copuestos antagonistas de acido ribonucleico para la modulacion de proproteina convertasa subtilisina/kexina tipo 9a.
EP2126079A1 (en) * 2007-03-22 2009-12-02 Santaris Pharma A/S Rna antagonist compounds for the inhibition of apo-b100 expression
JP2010525826A (ja) * 2007-05-03 2010-07-29 ロゼッタ インファーマティックス エルエルシー 癌を治療するためのmir34治療剤を含む組成物
DE102007021870A1 (de) 2007-05-10 2008-11-20 Clariant International Limited Wässrige Pigmentpräparationen
EP2198024A2 (en) 2007-08-30 2010-06-23 Santaris Pharma A/S Rna antagonist compounds for the modulation of fabp4/ap2
WO2010093788A2 (en) * 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
EP2563922A1 (en) * 2010-04-26 2013-03-06 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
WO2012144906A1 (en) * 2011-04-22 2012-10-26 Prosensa Technologies B.V. New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1)
EP2791335B1 (en) * 2011-12-16 2018-11-14 National University Corporation Tokyo Medical and Dental University Chimeric double-stranded nucleic acid

Similar Documents

Publication Publication Date Title
JP2016526529A5 (enExample)
JP2016528887A5 (enExample)
JP2017502668A5 (enExample)
JP2015133993A5 (enExample)
JP2016195614A5 (enExample)
HUE057725T2 (hu) Módosított nukleozidok, nukleotidok és nukleinsavak és ezek felhasználása
JP2016525888A5 (enExample)
JP2019515674A5 (enExample)
WO2018041973A9 (en) Chemically modified single-stranded rna-editing oligonucleotides
CN106573011A8 (zh) 取代的核苷、核苷酸和其类似物
JP2012070736A5 (enExample)
CN111741967A (zh) 核苷类似物、制备方法及应用
ZA201901061B (en) 2'-substituted-n6-substituted purine nucleotides for rna virus treatment
EP3192869A4 (en) Isolated oligonucleotide and use thereof in nucleic acid sequencing
EP2791160A4 (en) NUCLEOSIDE, NUCLEOTIDE, AND MODIFIED NUCLEIC ACID COMPOSITIONS
MX381121B (es) Supresión del gen de la huntingtina inducida por la arni
ES2679373T3 (es) Nuevos ligandos de RIG-I y métodos para producirlos
EA201792663A1 (ru) Способы скрининга бактерий, архей, водорослей и дрожжей с использованием нуклеиновых кислот crispr
HUE063813T2 (hu) CRISPR hibrid DNS/RNS polinukleotidok és alkalmazási eljárások
MX379233B (es) Métodos y composiciones para el perfilado de adn.
ME03502B (me) Supstituisani nukleozidi, nukleotidi i njihovi analozi
LT3265102T (lt) Beta-d-2'-deoksi-2'alfa-fluor-2'-beta-c-pakeistieji-2-modifikuoti-n6- pakeistieji purino nukleotidai, skirti hcv gydymui
EP3146075A4 (en) Ion sensor dna and rna sequencing by synthesis using nucleotide reversible terminators
MY196912A (en) Short pendant arm linkers for nucleotides in sequencing applications
MX374553B (es) Derivado de ácido nucleico que tiene actividad inmunoestimuladora.