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
JP6604544B2 (ja
JP2016526529A (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 (https=) 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 (https=)
EP (1) EP3010514B1 (https=)
JP (1) JP6604544B2 (https=)
CN (1) CN105324119A (https=)
AU (1) AU2014282666A1 (https=)
CA (1) CA2915443A1 (https=)
WO (1) WO2014203518A1 (https=)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
AU2012284265B2 (en) 2011-07-19 2017-08-17 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
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
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
SG11201500239VA (en) 2012-07-13 2015-03-30 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
US10676737B2 (en) 2014-12-17 2020-06-09 Proqr Therapeutics Ii B.V. Targeted RNA editing
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
EP3353306A4 (en) 2015-09-25 2019-06-05 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
JP7091255B2 (ja) * 2016-05-06 2022-06-27 アイオニス・ファーマシューティカルズ・インコーポレイテッド Ｇｌｐ－１受容体リガンド部分コンジュゲート化オリゴヌクレオチドおよびその使用
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
CA3029772A1 (en) * 2016-07-05 2018-01-11 Biomarin Technologies B.V. Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
EP3517610A4 (en) 2016-09-23 2020-05-27 National University Corporation Tokyo Medical and Dental University THROUGH HETERODUPLEX NUCLEIC ACID THROUGH BLOOD BRAIN CABINET
US11260134B2 (en) 2016-09-29 2022-03-01 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
WO2019004420A1 (ja) 2017-06-30 2019-01-03 国立大学法人東京医科歯科大学 ヘテロ二本鎖型ａｎｔｉｍｉＲ
US20200268898A1 (en) * 2017-11-08 2020-08-27 Astrazeneca Ab Glp-1 receptor ligand moiety conjugated oligonucleotides and uses thereof
JP7360170B2 (ja) 2018-02-28 2023-10-12 国立大学法人 東京医科歯科大学 虚血病変部位特異的な遺伝子治療法
EP3766972B1 (en) 2018-03-14 2025-04-23 Institute of Science Tokyo Nucleic acid complex
US11851654B2 (en) 2018-03-19 2023-12-26 National University Corporation Tokyo Medical And Dental University Nucleic acid with reduced toxicity
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
CN113614232A (zh) * 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
EP3954395A4 (en) * 2019-04-08 2023-06-28 National University Corporation Tokyo Medical and Dental University Pharmaceutical composition for muscle disease treatment
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
JP7793144B2 (ja) * 2019-10-11 2026-01-05 国立大学法人東京科学大学 修飾ヘテロ核酸
EP4090744A4 (en) * 2020-01-17 2024-04-10 Anastasia Khvorova UNIVERSAL DYNAMIC PHARMACOKINETIC MODIFYING ANCHORS
US20230174981A1 (en) * 2020-03-16 2023-06-08 National University Corporation Tokyo Medical And Dental University Heteronucleic acid containing morpholino nucleic acid
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
WO2022106695A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
AU2022299169A1 (en) 2021-06-23 2024-02-08 Beth Israel Deaconess Medical Center, Inc. Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
US20250339459A1 (en) * 2021-08-19 2025-11-06 National University Corporation Tokyo Medical And Dental University Modified heteronucleic acid containing morpholino nucleic acid
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
ES2192672T3 (es) 1996-11-18 2003-10-16 Takeshi Imanishi Nuevos analogos de nucleotidos.
JP3781879B2 (ja) 1996-11-18 2006-05-31 武 今西 新規ヌクレオチド類縁体
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
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US20010024808A1 (en) * 1998-09-10 2001-09-27 Millennium Pharmaceuticals, Inc., A Delaware Corporation Leptin induced genes
WO2001055455A2 (en) * 2000-01-31 2001-08-02 Millennium Pharmaceuticals, Inc. Resistance sequences and uses thereof
ATE433489T1 (de) * 2000-03-31 2009-06-15 Millennium Pharm Inc 16836, ein mitglied der humanen phospholipase c familie und seine verwendungen
CA2504694C (en) * 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP2336318B1 (en) 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
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
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
ES2603379T3 (es) 2006-10-09 2017-02-27 Roche Innovation Center Copenhagen A/S Compuestos antagonistas de ARN para la modulación de PCSK9
CA2681406A1 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S Rna antagonist compounds for the inhibition of apo-b100 expression
WO2008137862A2 (en) * 2007-05-03 2008-11-13 Rosetta Inpharmatics Llc Methods of using mir34 as a biomarker for tp53 functional status
DE102007021870A1 (de) 2007-05-10 2008-11-20 Clariant International Limited Wässrige Pigmentpräparationen
WO2009027527A2 (en) * 2007-08-30 2009-03-05 Santaris Pharma A/S Rna antagonist compounds for the modulation of fabp4/ap2
US20100249214A1 (en) * 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
US20130190383A1 (en) * 2010-04-26 2013-07-25 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
JP2014513946A (ja) * 2011-04-22 2014-06-19 プロセンサ テクノロジーズ ビー．ブイ． 筋強直性ジストロフィー１型（ｄｍ１）などのヒト遺伝性疾患の治療、遅延及び／又は予防のための新規の化合物
AU2012353330B2 (en) * 2011-12-16 2018-04-19 National University Corporation Tokyo Medical And Dental University Chimeric double-stranded nucleic acid

Similar Documents

Publication Publication Date Title
JP2016526529A5 (https=)
JP2016528887A5 (https=)
JP2017502668A5 (https=)
JP2015133993A5 (https=)
JP2016195614A5 (https=)
HUE057725T2 (hu) Módosított nukleozidok, nukleotidok és nukleinsavak és ezek felhasználása
JP2016525888A5 (https=)
JP2019515674A5 (https=)
WO2018041973A9 (en) Chemically modified single-stranded rna-editing oligonucleotides
CN106573011A8 (zh) 取代的核苷、核苷酸和其类似物
EP4328322A3 (en) Probe library construction
EP3192869A4 (en) Isolated oligonucleotide and use thereof in nucleic acid sequencing
EP2791160A4 (en) NUCLEOSIDE, NUCLEOTIDE, AND MODIFIED NUCLEIC ACID COMPOSITIONS
ZA201901061B (en) 2'-substituted-n6-substituted purine nucleotides for rna virus treatment
ES2679373T3 (es) Nuevos ligandos de RIG-I y métodos para producirlos
MX381121B (es) Supresión del gen de la huntingtina inducida por la arni
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
IL231460B (en) 5 - methoxy, ho - '3, unblocked nucleotides that undergo rapid degradation by light and methods for nucleic acid sequencing
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.
EP3381947A4 (en) DNA Polymerase VARIANT