JP7506601B2 - 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法 - Google Patents

筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法 Download PDF

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JP7506601B2
JP7506601B2 JP2020528393A JP2020528393A JP7506601B2 JP 7506601 B2 JP7506601 B2 JP 7506601B2 JP 2020528393 A JP2020528393 A JP 2020528393A JP 2020528393 A JP2020528393 A JP 2020528393A JP 7506601 B2 JP7506601 B2 JP 7506601B2
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polynucleic acid
acid molecule
nucleotides
sirna molecule
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JP2021507686A (ja
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ジョン ゲル,アンドリュー
ラマナ ドパラプディ,ヴェンカタ
サイ-ホ チュウ,デイヴィッド
カラミアン コクラン,マイケル
フード,マイケル
ダイアナ ダリモント,ベアトリス
バーク,ロブ
シ,ユンユ
エルドガン マレリウス,グリン
マレコヴァ,バーボラ
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アビディティー バイオサイエンシーズ,インク.
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JP2020528393A 2017-12-06 2018-12-06 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法 Active JP7506601B2 (ja)

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JP2022162646A JP7564852B2 (ja) 2017-12-06 2022-10-07 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
JP2024169131A JP2025000818A (ja) 2017-12-06 2024-09-27 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法

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US201762595545P 2017-12-06 2017-12-06
US62/595,545 2017-12-06
US201862725883P 2018-08-31 2018-08-31
US62/725,883 2018-08-31
PCT/US2018/064359 WO2019113393A1 (en) 2017-12-06 2018-12-06 Compositions and methods of treating muscle atrophy and myotonic dystrophy

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JP2022162646A Active JP7564852B2 (ja) 2017-12-06 2022-10-07 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
JP2024169131A Pending JP2025000818A (ja) 2017-12-06 2024-09-27 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法

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JP2024169131A Pending JP2025000818A (ja) 2017-12-06 2024-09-27 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法

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Families Citing this family (64)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
CN118667812A (zh) 2017-12-06 2024-09-20 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
US20210095283A1 (en) * 2018-01-04 2021-04-01 Avidity Biosciences, Inc. Heteroduplex nucleic acid molecules and uses thereof
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
JP2021533200A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2020028836A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
IL319265A (en) 2018-12-21 2025-04-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and their uses
JP7592034B2 (ja) 2019-06-06 2024-11-29 アビディティー バイオサイエンシーズ,インク. 核酸ポリペプチド組成物およびその使用
KR20220019259A (ko) 2019-06-06 2022-02-16 어비디티 바이오사이언시스 인크. Una 아미다이트 및 이의 용도
AU2020311399A1 (en) * 2019-07-10 2022-02-17 University Of Washington Non-viral immuno-targeting
US20220267778A1 (en) * 2019-07-30 2022-08-25 Shionogi & Co., Ltd. Nucleic acid drug targeting murf1
JP2022545265A (ja) * 2019-08-20 2022-10-26 ニューリタス リミテッド 筋萎縮を治療するためのペプチド
WO2021076856A1 (en) * 2019-10-18 2021-04-22 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2021142217A1 (en) * 2020-01-10 2021-07-15 Dyne Thereapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of milck1
CA3163608A1 (en) * 2020-01-10 2021-07-15 Romesh R. Subramanian Muscle-targeting complexes and uses thereof
US20230088865A1 (en) * 2020-01-10 2023-03-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
BR112022013686A2 (pt) * 2020-01-10 2022-09-06 Dyne Therapeutics Inc Complexos de direcionamento a músculo e usos dos mesmos para tratar distrofia miotônica
WO2021142269A1 (en) * 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of genes associated with muscle atrophy
CN115335062A (zh) * 2020-01-10 2022-11-11 达因疗法公司 肌肉靶向复合物及其用于调节与肌肉健康相关的基因的用途
EP4087878A4 (en) * 2020-01-10 2024-06-05 Dyne Therapeutics, Inc. MUSCLE-TARGETED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES
WO2021142331A1 (en) * 2020-01-10 2021-07-15 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for modulation of genes associated with cardiac muscle disease
WO2021150382A1 (en) * 2020-01-24 2021-07-29 Dyne Therapeutics, Inc. Muscle-targeting complexes and uses thereof in treating muscle atrophy
MX2022009418A (es) * 2020-01-31 2022-08-25 Dyne Therapeutics Inc Anticuerpo del receptor anti-transferrina (tfr) y usos del mismo.
GEP20257742B (en) * 2020-03-06 2025-03-10 Aligos Therapeutics Inc Modified short interfering nucleic acid (sina) molecules and uses thereof
EP4121063A4 (en) 2020-03-19 2024-07-03 Avidity Biosciences, Inc. COMPOSITIONS AND METHODS FOR TREATING FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
JP7748962B2 (ja) 2020-03-27 2025-10-03 アビディティー バイオサイエンシーズ,インク. 筋ジストロフィーを処置するための組成物および方法
EP4146229A4 (en) * 2020-05-05 2025-07-23 Avidity Biosciences Inc COMPOSITIONS AND METHODS FOR TREATING POMPE DISEASE
EP4185320A4 (en) * 2020-07-23 2025-10-08 Dyne Therapeutics Inc MUSCLE TARGETING COMPLEXES AND THEIR USES IN THE TREATMENT OF MUSCLE ATROPHY
JP2023535078A (ja) * 2020-07-23 2023-08-15 ダイン セラピューティクス,インコーポレーテッド 筋緊張性ジストロフィーを処置するための筋標的化複合体およびその使用
WO2022056266A2 (en) * 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
EP4271816A4 (en) * 2020-12-31 2025-04-02 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND RELATED USES FOR THE TREATMENT OF MYOTONIC DYSTROPHY
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11648318B2 (en) 2021-07-09 2023-05-16 Dyne Therapeutics, Inc. Anti-transferrin receptor (TFR) antibody and uses thereof
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11827702B2 (en) 2021-09-01 2023-11-28 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof
US11833221B2 (en) * 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
KR20240055874A (ko) 2021-09-16 2024-04-29 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증을 치료하는 조성물 및 방법
EP4209220A4 (en) 2021-11-23 2024-08-28 Advanced Protein Technologies Corp. COMPOSITION FOR IMPROVING, PREVENTING OR TREATING MUSCLE ATROPHY OR SARCOPENIA, CONTAINING 2'-FL
AU2021476277A1 (en) * 2021-12-02 2024-06-06 Linno Pharmaceuticals Inc. Transferrin binding antibodies and use thereof
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
KR20250004770A (ko) 2022-04-15 2025-01-08 다인 세라퓨틱스, 인크. 근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 제제
WO2024063570A1 (ko) * 2022-09-23 2024-03-28 주식회사 셀리버리 근긴장성 이영양증 단백질 키나아제를 표적화하는 세포투과성 펩티드 융합 안티센스 올리고뉴클레오티드 및 이의 용도
CN120475978A (zh) * 2022-10-19 2025-08-12 箭头药业股份有限公司 用于抑制DM1蛋白激酶(DMPK)表达的RNAi剂、其组合物和使用方法
WO2024187057A2 (en) 2023-03-08 2024-09-12 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof
US12491257B2 (en) 2023-06-27 2025-12-09 Avidity Biosciences, Inc. Compositions and methods of using PRKAG2-targeting antibody-oligonucleotide conjugates
US12409230B2 (en) 2023-06-30 2025-09-09 Avidity Biosciences, Inc. Compositions comprising PLN-targeting anti-transferrin receptor antibody-polynucleotides and methods of use thereof to treat cardiomyopathy
US20250064958A1 (en) * 2023-08-22 2025-02-27 Eli Lilly And Company AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENTS
WO2025072685A1 (en) * 2023-09-28 2025-04-03 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
TW202517685A (zh) 2023-10-26 2025-05-01 美商百健Ma公司 抗運鐵蛋白受體抗體及其用途
WO2025147541A1 (en) * 2024-01-02 2025-07-10 Dyne Therapeutics, Inc. Methods and compositions for treating myotonic dystrophy
CN120648679A (zh) * 2024-03-15 2025-09-16 迦进生物医药(上海)有限公司 抑制dmpk表达的多核苷酸分子及其用途
WO2025207959A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Anti-transferrin receptor antibodies and uses thereof
CN119391706B (zh) * 2024-12-31 2025-04-25 北京尧景基因技术有限公司 抑制MSTN基因表达的siRNA及其缀合物和应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002537318A (ja) 1999-02-22 2002-11-05 ジョージタウン・ユニバーシティ 全身性遺伝子送達のための抗体標的化フラグメントイムノリポソーム
WO2013138662A1 (en) 2012-03-16 2013-09-19 4S3 Bioscience, Inc. Antisense conjugates for decreasing expression of dmpk
JP2013538560A (ja) 2010-07-19 2013-10-17 シー・フランク・ベネット 筋緊張性ジストロフィー・プロテインキナーゼ(dmpk)発現の調整
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping

Family Cites Families (210)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1000075A (en) 1910-07-27 1911-08-08 William R Collins Tunneling-machine.
US4694778A (en) 1984-05-04 1987-09-22 Anicon, Inc. Chemical vapor deposition wafer boat
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
DE3687030T2 (de) 1985-03-15 1993-03-11 Eugene Stirchak Stereoregulare polynukleotiden bindende polymere.
US4828971A (en) 1988-03-24 1989-05-09 Eastman Kodak Company Thermally processable element comprising a backing layer
CA1251835A (en) 1988-04-05 1989-03-28 Wai-Cheung Tang Dielectric image-resonator multiplexer
WO1991004753A1 (en) 1989-10-02 1991-04-18 Cetus Corporation Conjugates of antisense oligonucleotides and therapeutic uses thereof
DE69123979T2 (de) 1990-10-12 1997-04-30 Max Planck Gesellschaft Abgeänderte ribozyme
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
KR100361933B1 (ko) 1993-09-08 2003-02-14 라 졸라 파마슈티칼 컴파니 화학적으로정의된비중합성결합가플랫폼분자및그것의콘주게이트
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
AU691306B2 (en) 1994-06-17 1998-05-14 F. Hoffmann-La Roche Ag N,n'-bis(quinolin-4-yl)-diamine derivatives, their preparation and their use as antimalarials
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
ATE373672T1 (de) 1996-01-16 2007-10-15 Sirna Therapeutics Inc Synthese von methoxynukleoside und enzymatische nukleisäure moleküle
WO1997034631A1 (en) 1996-03-18 1997-09-25 Board Of Regents, The University Of Texas System Immunoglobin-like domains with increased half lives
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US6635452B1 (en) 1996-12-10 2003-10-21 Sequenom Inc. Releasable nonvolatile mass label molecules
US20030073207A1 (en) 1997-01-31 2003-04-17 Saghir Akhtar Enzymatic nucleic acid treatment of diseases or conditions related to levels of epidermal growth factor receptors
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
DE69937291T2 (de) 1998-04-02 2008-07-10 Genentech, Inc., South San Francisco Antikörpervarianten und fragmente davon
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
EP1071753A2 (en) 1998-04-20 2001-01-31 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
GB9816575D0 (en) 1998-07-31 1998-09-30 Zeneca Ltd Novel compounds
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US6849272B1 (en) 1999-04-21 2005-02-01 Massachusetts Institute Of Technology Endosomolytic agents and cell delivery systems
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
DE10160151A1 (de) 2001-01-09 2003-06-26 Ribopharma Ag Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
CA2328356A1 (en) 1999-12-22 2001-06-22 Itty Atcravi Recreational vehicles
CA2395660A1 (en) 1999-12-29 2001-07-12 Immunogen, Inc. Cytotoxic agents comprising modified doxorubicins and daunorubicins and their therapeutic use
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
EP1309726B2 (en) 2000-03-30 2018-10-03 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
ES2410907T5 (es) 2000-03-30 2019-08-20 The Whitehead Institute For Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
JP2003533986A (ja) 2000-05-04 2003-11-18 エイブイアイ バイオファーマ, インコーポレイテッド スプライス領域アンチセンス組成物および方法
JP2002095476A (ja) * 2000-09-20 2002-04-02 Eisai Co Ltd トランスフェリン受容体を介した遺伝子導入法
RU2322500C2 (ru) 2000-12-01 2008-04-20 Макс-Планк-Гезелльшафт Цур Фердерунг Дер Виссеншафтен Е.Ф. Малые молекулы рнк, опосредующие интерференцию рнк
US8546143B2 (en) 2001-01-09 2013-10-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US6884869B2 (en) 2001-04-30 2005-04-26 Seattle Genetics, Inc. Pentapeptide compounds and uses related thereto
DE10133858A1 (de) 2001-07-12 2003-02-06 Aventis Pharma Gmbh Synthetische doppelsträngige Oligonucleotide zur gezielten Hemmung der Genexpression
US6942972B2 (en) 2001-10-24 2005-09-13 Beckman Coulter, Inc. Efficient synthesis of protein-oligonucleotide conjugates
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
ES2280826T5 (es) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Nuevas formas adicionales de moléculas de ARN de interferencia
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
DK2284266T3 (da) 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US8084582B2 (en) 2003-03-03 2011-12-27 Xencor, Inc. Optimized anti-CD20 monoclonal antibodies having Fc variants
JP4605799B2 (ja) 2003-04-02 2011-01-05 ダーマコン, インコーポレイテッド Rna干渉において使用するための修飾ポリヌクレオチド
WO2004100987A2 (en) * 2003-05-06 2004-11-25 Regeneron Pharmaceuticals, Inc. Methods of using il-1 antagonists to treat neointimal hyperplasia
ATE485394T1 (de) 2003-06-02 2010-11-15 Univ Massachusetts Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
AU2004248136B2 (en) 2003-06-02 2011-09-15 University Of Massachusetts Methods and compositions for controlling efficacy of RNA silencing
EP2336317B1 (en) 2003-06-13 2019-09-11 Alnylam Europe AG Double-stranded ribonucleic acid with increased effectiveness in an organism
US8133733B2 (en) 2003-10-24 2012-03-13 Gencia Corporation Nonviral vectors for delivering polynucleotides to target tissues
NZ583292A (en) 2003-11-06 2012-03-30 Seattle Genetics Inc Monomethylvaline compounds capable of conjugation to ligands
US7494925B2 (en) 2004-02-23 2009-02-24 Micron Technology, Inc. Method for making through-hole conductors for semiconductor substrates
US8389710B2 (en) 2004-02-27 2013-03-05 Operational Technologies Corporation Therapeutic nucleic acid-3′-conjugates
JP5243789B2 (ja) 2004-03-15 2013-07-24 シティ・オブ・ホープ 二本鎖rnaによる遺伝子発現の特異的阻害のための方法及び組成物
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
EP1765847A4 (en) 2004-05-27 2010-10-20 Alnylam Pharmaceuticals Inc NUCLEASERESISTENT DOUBLE-STRANDED RIBONUCLEIC ACID
ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
AU2005332660A1 (en) 2004-11-12 2006-12-14 Seattle Genetics, Inc. Auristatins having an aminobenzoic acid unit at the N terminus
US7429482B2 (en) * 2005-01-13 2008-09-30 United States Of America As Represented By The Department Of Veterans Affairs Screening tools for discovery of novel anabolic agents
CA2597265C (en) 2005-02-10 2015-03-24 Baylor Research Institute Anti-interferon alpha monoclonal antibodies and methods for use
AU2006249340A1 (en) 2005-05-26 2006-11-30 Ensemble Discovery Corportion Biodetection by nucleic acid-templated chemistry
US20070054259A1 (en) * 2005-06-17 2007-03-08 City Of Hope Modulation of hnRNP H and treatment of DM1
EP1915449B1 (en) 2005-08-17 2016-03-23 Bioneer Corporation Sirna-hydrophilic polymer conjugates for intracellular delivery of sirna and method thereof
WO2007028065A2 (en) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007121272A2 (en) * 2006-04-11 2007-10-25 The Board Of Regents Of The University Of Texas System Methods and compositions involving nucleotide repeat disorders
ES2645410T3 (es) 2006-05-10 2017-12-05 Sarepta Therapeutics, Inc. Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
US7521232B2 (en) 2006-05-31 2009-04-21 Icx Nomadics, Inc. Emissive species for clinical imaging
AU2007254853B2 (en) 2006-06-02 2011-11-17 Aveo Pharmaceuticals, Inc. Hepatocyte growth factor (HGF) binding proteins
JP2009542810A (ja) 2006-07-10 2009-12-03 バイオジェン・アイデック・エムエイ・インコーポレイテッド Smad4欠損癌の増殖を阻害するための組成物および方法
WO2008018795A1 (en) 2006-08-11 2008-02-14 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
BRPI0715375A2 (pt) 2006-08-18 2013-06-18 Hoffmann La Roche policonjugados para distribuiÇço in vivo de polinucleotÍdeos
ES2873350T3 (es) 2007-08-27 2021-11-03 1Globe Health Inst Llc Composiciones de ARN interferente asimétrico y usos de las mismas
WO2009026933A1 (en) 2007-08-29 2009-03-05 Vestas Wind Systems A/S Monopile foundation for offshore wind turbine
JP2010538678A (ja) 2007-09-18 2010-12-16 イントラドイグム コーポレーション K−rassiRNA含有組成物及びそれらの使用法
DK2195428T3 (en) 2007-09-19 2014-03-03 Applied Biosystems Llc SIRNA SEQUENCE-INDEPENDENT MODIFICATION FORMS TO REDUCE TARGET-FAILING PHENOTYPIC EFFECTS OF RNAI, AND STABILIZED FORMS THEREOF
PT2203173E (pt) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
MX2010007767A (es) 2008-01-18 2010-08-09 Medimmune Llc Anticuerpos manipulados con cisteina para conjugacion especifica de sitio.
US20110065774A1 (en) 2008-01-31 2011-03-17 Alnylam Pharmaceuticals Chemically modified oligonucleotides and uses thereof
US20110081362A1 (en) 2008-01-31 2011-04-07 The Brigham And Women's Hospital, Inc. Treatment of cancer
AU2009209565B2 (en) 2008-02-01 2013-09-19 Ascendis Pharma As Prodrug comprising a self-cleavable linker
EP3643782A1 (en) 2008-02-11 2020-04-29 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
DK2842575T3 (da) 2008-03-18 2017-11-27 Seattle Genetics Inc Auristatin-lægemiddel-linker-konjugater
AU2009234266B2 (en) 2008-04-11 2015-08-06 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
JP5667043B2 (ja) 2008-04-15 2015-02-12 アメリカ合衆国GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTHAND HUMAN SERVICES 阻害性オリゴヌクレオチドを送達するための組成物および方法
US8248352B2 (en) 2008-04-25 2012-08-21 Lg Display Co., Ltd. Driving circuit of liquid crystal display
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
EP2657342B1 (en) 2008-08-08 2015-11-18 Genisphere, LLC DNA dendrimers protected against nuclease degradation
WO2010019718A2 (en) 2008-08-13 2010-02-18 California Institute Of Technology Carrier nanoparticles and related compositions, methods and systems
EP2690175B1 (en) 2008-09-02 2016-12-28 Alnylam Pharmaceuticals, Inc. Compositions and methods for combined inhibition of mutant EGFR gene and IL-6 expression
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
CN108165548B (zh) 2008-09-22 2022-10-14 菲奥医药公司 减小大小的自递送RNAi化合物
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
DK2344637T4 (en) 2008-10-27 2018-04-23 Biomarin Tech Bv Methods and means for efficiently skipping exon 45 in Duchenne muscular dystrophy pre-mRNA
KR101602431B1 (ko) 2008-11-05 2016-03-10 삼성전자 주식회사 냉장고
RU2015151857A (ru) 2008-12-02 2019-01-15 Уэйв Лайф Сайенсес Джапан, Инк. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
US8481698B2 (en) 2009-03-19 2013-07-09 The President And Fellows Of Harvard College Parallel proximity ligation event analysis
WO2010115033A2 (en) 2009-04-02 2010-10-07 Regents Of The University Of Minnesota Nucleotide repeat expansion-associated polypeptides and uses thereof
NZ595955A (en) 2009-04-24 2012-10-26 Prosensa Technologies Bv Oligonucleotide comprising an inosine for treating dmd
BRPI1011269A2 (pt) 2009-05-05 2016-09-27 Altermune Technologies Llc imunidade quimicamente programável
MX338775B (es) 2009-06-04 2016-05-02 Novartis Ag Metodos para identificacion de sitios para conjugacion de igg.
US20120213705A1 (en) 2009-06-22 2012-08-23 Medimmune, Llc ENGINEERED Fc REGIONS FOR SITE-SPECIFIC CONJUGATION
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2011003780A1 (en) 2009-07-06 2011-01-13 F. Hoffmann-La Roche Ag Bi-specific digoxigenin binding antibodies
WO2011009624A1 (en) 2009-07-22 2011-01-27 Cenix Bioscience Gmbh Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes
EP2473607A2 (en) 2009-08-31 2012-07-11 INSERM - Institut National de la Santé et de la Recherche Médicale Exon skipping therapy for functional amelioration of semi functional dystrophin in becker and duchenne muscular dystrophy
KR101808602B1 (ko) 2009-10-07 2017-12-13 마크로제닉스, 인크. 푸코실화 정도의 변경으로 인해 개선된 이펙터 기능을 나타내는 Fc 영역-함유 폴리펩티드, 그것의 사용 방법
CA2780563C (en) 2009-11-12 2025-05-06 Univ Western Australia Antisense Molecules and Methods for Treating Pathologies
EP2534174A4 (en) 2010-02-12 2015-02-25 Solulink Inc Preparation and / or purification of oligonucleotide conjugates
CN102971329B (zh) 2010-04-15 2016-06-29 麦迪穆有限责任公司 用于治疗增殖性疾病的吡咯并苯并二氮杂卓
NZ602932A (en) 2010-04-15 2014-08-29 Seattle Genetics Inc Targeted pyrrolobenzodiazapine conjugates
EP3173419A1 (en) 2010-04-28 2017-05-31 Ionis Pharmaceuticals, Inc. Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
EP2576574A2 (en) 2010-05-28 2013-04-10 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
EP2580326A1 (en) 2010-06-11 2013-04-17 Antisense Pharma GmbH Method for selective oligonucleotide modification
CN103068853B (zh) 2010-08-19 2016-08-10 Peg生物制药公司 协同性生物分子-聚合物轭合物
US9260471B2 (en) 2010-10-29 2016-02-16 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
MX347298B (es) 2010-12-29 2017-04-21 Arrowhead Res Corp Conjugados de suministro de polinucleotidos in vivo que tienen acoplamientos sensibles a enzima.
WO2012109296A1 (en) 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
US9045750B2 (en) 2011-03-18 2015-06-02 Yuelong Ma Humanized lewis-Y specific antibody-based delivery of dicer substrate siRNA (D-siRNA) against STAT3
KR20160044598A (ko) 2011-03-29 2016-04-25 로슈 글리카트 아게 항체 Fc 변이체
US10100305B2 (en) 2011-07-15 2018-10-16 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation of start sites
EP3248982A1 (en) 2011-07-19 2017-11-29 Wave Life Sciences Ltd. Thiosulfonate reagents for the synthesis of functionalized nucleic acids
US9418216B2 (en) 2011-07-21 2016-08-16 Microsoft Technology Licensing, Llc Cloud service authentication
EP2751270B1 (en) 2011-08-29 2018-08-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
JP6393617B2 (ja) 2011-10-14 2018-09-19 シアトル ジェネティクス,インコーポレーテッド ピロロベンゾジアゼピンおよび標的コンジュゲート
WO2013071079A1 (en) 2011-11-09 2013-05-16 President And Fellows Of Harvard College Lin28/let-7 crystal structures, purification protocols, and molecular probes suitable for screening assays and therapeutics
KR20250133484A (ko) 2011-11-18 2025-09-05 알닐람 파마슈티칼스 인코포레이티드 변형된 RNAi 제제
GB201121069D0 (en) * 2011-12-07 2012-01-18 Isis Innovation Delivery system
WO2013093809A1 (en) 2011-12-23 2013-06-27 Pfizer Inc. Engineered antibody constant regions for site-specific conjugation and methods and uses therefor
TW201333040A (zh) 2012-01-06 2013-08-16 Bioalliance Cv 抗-輸鐵蛋白受器之抗體及其使用方法
EP2814512A1 (en) 2012-02-16 2014-12-24 UCL Business Plc. Lysosome-cleavable linker
EP2818480B1 (en) 2012-02-24 2020-08-26 Alteogen Inc. Modified antibody in which motif comprising cysteine residue is bound, modified antibody-drug conjugate comprising the modified antibody, and production method for same
AR090905A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
CN109481455A (zh) 2012-05-02 2019-03-19 箭头研究公司 治疗kras相关疾病的有机组合物
EP2849797B1 (en) 2012-05-15 2019-12-18 Concortis Biosystems, Corp Drug-conjugates, conjugation methods, and uses thereof
US9504756B2 (en) 2012-05-15 2016-11-29 Seattle Genetics, Inc. Self-stabilizing linker conjugates
CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
CA2880869A1 (en) 2012-08-20 2014-02-27 The Regents Of The University Of California Polynucleotides having bioreversible groups
HUE045435T2 (hu) 2012-10-12 2019-12-30 Medimmune Ltd Pirrolobenzodiazepinek és konjugátumaik
SG11201503821YA (en) 2012-11-15 2015-06-29 Roche Innovation Ct Copenhagen As Oligonucleotide conjugates
EP2922818B1 (en) 2012-11-24 2018-09-05 Hangzhou Dac Biotech Co., Ltd Hydrophilic linkers and their uses for conjugation of drugs to cell binding molecules
US9089614B2 (en) 2012-12-21 2015-07-28 Bioalliance C.V. Hydrophilic self-immolative linkers and conjugates thereof
US9481905B2 (en) 2013-02-19 2016-11-01 Orizhan Bioscience Limited Method of using neutrilized DNA (N-DNA) as surface probe for high throughput detection platform
US9319764B2 (en) 2013-03-08 2016-04-19 Merry Electronics Co., Ltd. MEMS microphone packaging structure
NZ775701A (en) 2013-03-14 2022-08-26 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
KR102306490B1 (ko) 2013-03-15 2021-09-28 리제너론 파아마슈티컬스, 인크. 생물학적 활성 분자, 그의 접합체 및 치료 용도
US9577459B2 (en) 2013-03-15 2017-02-21 Sequana Medical Ag Systems and methods for regulating inductive energy transfer to an implantable system
NZ732507A (en) 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
EP2777714A1 (en) 2013-03-15 2014-09-17 NBE-Therapeutics LLC Method of producing an immunoligand/payload conjugate by means of a sequence-specific transpeptidase enzyme
CA2908096C (en) 2013-03-27 2022-05-03 Isarna Therapeutics Gmbh Modified tgf-beta oligonucleotides
US9295731B2 (en) 2013-04-01 2016-03-29 Mark Quang Nguyen Cleavable drug conjugates, compositions thereof and methods of use
WO2014169049A1 (en) 2013-04-09 2014-10-16 Duke University 2' fluoro-modified rnas as immunostimulators
US20180104349A9 (en) 2013-04-28 2018-04-19 Gang Qin Novel linker, preparation method, and application thereof
EP4324480A3 (en) * 2013-05-20 2024-05-08 F. Hoffmann-La Roche AG Anti-transferrin receptor antibodies and methods of use
CA2913370C (en) 2013-05-31 2022-12-13 Zymeworks Inc. Heteromultimers with reduced or silenced effector function
US11229711B2 (en) 2013-06-06 2022-01-25 Magenta Therapeutics, Inc. Linkers for antibody-drug conjugates and related compounds, compositions, and methods of use
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
EP2845607A1 (en) 2013-09-09 2015-03-11 University of Vienna Antisense oligonucleotides with improved pharmacokinetic properties
WO2015038426A1 (en) 2013-09-13 2015-03-19 Asana Biosciences, Llc Self-immolative linkers containing mandelic acid derivatives, drug-ligand conjugates for targeted therapies and uses thereof
BR112016007622A2 (pt) 2013-10-15 2018-01-23 Seattle Genetics, Inc. composto, composição farmacêutica, conjugado de ligante-droga, e, método para tratar câncer
WO2015057876A1 (en) 2013-10-15 2015-04-23 Sorrento Therapeutics Inc. Drug-conjugates with a targeting molecule and two different drugs
EP3610884B1 (en) 2013-11-06 2021-08-18 Merck Sharp & Dohme Corp. Peptide containing conjugates for dual molecular delivery of oligonucleotides
US10378017B2 (en) 2013-12-02 2019-08-13 Brandeis University High temperature selection of nucleotide-supported carbohydrate vaccines and resulting glycosylated oligonucleotides
MX2016009290A (es) 2014-01-16 2017-02-28 Wave Life Sciences Ltd Diseño quiral.
CA2935426C (en) 2014-01-30 2023-07-25 F. Hoffmann-La Roche Ag Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target
EP3159409B1 (en) 2014-06-17 2019-12-04 Nippon Shinyaku Co., Ltd. Antisense nucleic acid for use in the treatment of duchenne's muscular dystrophy
TWI695011B (zh) 2014-06-18 2020-06-01 美商梅爾莎納醫療公司 抗her2表位之單株抗體及其使用之方法
EP3158067B1 (en) 2014-06-23 2020-08-12 Monsanto Technology LLC Compositions and methods for regulating gene expression via rna interference
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
JP2018512876A (ja) 2015-04-22 2018-05-24 ミナ セラピューティクス リミテッド saRNA組成物および使用方法
WO2016179112A1 (en) 2015-05-01 2016-11-10 Precision Biosciences, Inc. Precise deletion of chromoscomal sequences in vivo and treatment of nucleotide repeat expansion disorders using engineered nucleases
NZ737757A (en) 2015-05-19 2023-07-28 Sarepta Therapeutics Inc Peptide oligonucleotide conjugates
IL302486A (en) 2015-06-24 2023-06-01 Hoffmann La Roche Antibodies against the transnephrine receptor with adapted affinity
JP2019517991A (ja) 2016-03-01 2019-06-27 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト 低減したadcpを有するオビヌツズマブ及びリツキシマブ変異体
MA45328A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
CN109310765A (zh) 2016-06-20 2019-02-05 领先基因生物技术股份有限公司 抗体-药物偶联物
US11427838B2 (en) * 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
CA3039922A1 (en) 2016-10-28 2018-05-03 Genethon Compositions and methods for the treatment of myotonic dystrophy
CA3039733A1 (en) 2016-10-28 2018-05-03 Genethon Compositions and methods for the treatment of myotonic dystrophy
KR20240035907A (ko) 2016-11-23 2024-03-18 알닐람 파마슈티칼스 인코포레이티드 표적외 효과가 감소된 변형 rna 작용제
IT201700008383A1 (it) 2017-01-26 2018-07-26 Explea S R L Dispositivo per la produzione di energia elettrica utilizzante l’azione combinata di campi magnetici pulsati e di correnti fluidodinamiche agenti su una lamina flessibile dotata di un elemento piezoelettrico ed impianto formato da più elementi modulari collegati elettricamente fra loro e composti ciascuno da una pluralità di tali dispositivi
KR20200004861A (ko) 2017-05-05 2020-01-14 퓨전 파마슈티칼즈 인크. Igf-1r 모노클로날 항체 및 그의 용도
KR20250065737A (ko) 2017-09-22 2025-05-13 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
CN118667812A (zh) 2017-12-06 2024-09-20 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
CN112930197A (zh) 2018-08-02 2021-06-08 达因疗法公司 肌肉靶向复合物及其用途
MX2021001283A (es) 2018-08-02 2021-07-15 Dyne Therapeutics Inc Complejos dirigidos al músculo y sus usos para tratar la distrofia miotónica.
JP7748962B2 (ja) 2020-03-27 2025-10-03 アビディティー バイオサイエンシーズ,インク. 筋ジストロフィーを処置するための組成物および方法

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002537318A (ja) 1999-02-22 2002-11-05 ジョージタウン・ユニバーシティ 全身性遺伝子送達のための抗体標的化フラグメントイムノリポソーム
JP2013538560A (ja) 2010-07-19 2013-10-17 シー・フランク・ベネット 筋緊張性ジストロフィー・プロテインキナーゼ(dmpk)発現の調整
WO2013138662A1 (en) 2012-03-16 2013-09-19 4S3 Bioscience, Inc. Antisense conjugates for decreasing expression of dmpk
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
International Journal of Pharmaceutics (1998) Vol.163, pp.63-71
Journal of Controlled Release (2016) Vol.237, pp.1-13
Nature (2012) Vol.488, pp.111-115, METHODS

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