JP2021090458A5 - - Google Patents

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JP2021090458A5
JP2021090458A5 JP2021037024A JP2021037024A JP2021090458A5 JP 2021090458 A5 JP2021090458 A5 JP 2021090458A5 JP 2021037024 A JP2021037024 A JP 2021037024A JP 2021037024 A JP2021037024 A JP 2021037024A JP 2021090458 A5 JP2021090458 A5 JP 2021090458A5
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pharmaceutical composition
seq
intron
moiety
exon
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JP2021037024A 2015-03-18 2021-03-09 ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除 Pending JP2021090458A (ja)

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US201562135048P 2015-03-18 2015-03-18
US62/135,048 2015-03-18
US201562162571P 2015-05-15 2015-05-15
US62/162,571 2015-05-15

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JP2021090458A JP2021090458A (ja) 2021-06-17
JP2021090458A5 true JP2021090458A5 (enExample) 2021-11-11

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JP2017548452A Active JP6942054B2 (ja) 2015-03-18 2016-03-18 ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除
JP2021037024A Pending JP2021090458A (ja) 2015-03-18 2021-03-09 ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除

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US (2) US11015200B2 (enExample)
EP (1) EP3271372A4 (enExample)
JP (2) JP6942054B2 (enExample)
BR (1) BR112017019971A2 (enExample)
MA (1) MA41795A (enExample)
WO (1) WO2016149659A2 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
JP6875684B2 (ja) * 2015-09-16 2021-05-26 日本新薬株式会社 筋萎縮症治療用アンチセンス核酸
KR102670349B1 (ko) * 2016-12-30 2024-05-29 올리패스 주식회사 펩타이드 핵산 유도체들에 의한 엑손 스키핑
US11572560B2 (en) 2018-10-03 2023-02-07 Massachusetts Institute Of Technology Splicing-dependent transcriptional gene silencing or activation
CN114728999A (zh) * 2019-09-05 2022-07-08 赛诺菲 含有核苷酸类似物的寡核苷酸
AU2021390998A1 (en) * 2020-12-03 2023-06-29 Kyungsangbukdo (Kyungsangbukdo Livestock Research Institute) Transgenic animal having modified myostatin gene
EP4299743A4 (en) * 2021-02-26 2025-01-29 KNC Laboratories Co., Ltd. NUCLEIC ACID MEDICINAL PRODUCT EXPRESSING A SPLICE VARIANT OF MYOSTATIN

Family Cites Families (160)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4426330A (en) 1981-07-20 1984-01-17 Lipid Specialties, Inc. Synthetic phospholipid compounds
US4534899A (en) 1981-07-20 1985-08-13 Lipid Specialties, Inc. Synthetic phospholipid compounds
AU5661386A (en) 1985-03-15 1986-10-13 Stirchak, E. Stereoregular polynucleotide-binding polymers
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5354844A (en) 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5108921A (en) 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
US5227170A (en) 1989-06-22 1993-07-13 Vestar, Inc. Encapsulation process
US5356633A (en) 1989-10-20 1994-10-18 Liposome Technology, Inc. Method of treatment of inflamed tissues
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5527528A (en) 1989-10-20 1996-06-18 Sequus Pharmaceuticals, Inc. Solid-tumor treatment method
US5580575A (en) 1989-12-22 1996-12-03 Imarx Pharmaceutical Corp. Therapeutic drug delivery systems
US5469854A (en) 1989-12-22 1995-11-28 Imarx Pharmaceutical Corp. Methods of preparing gas-filled liposomes
US5852188A (en) 1990-01-11 1998-12-22 Isis Pharmaceuticals, Inc. Oligonucleotides having chiral phosphorus linkages
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
JP3220180B2 (ja) 1991-05-23 2001-10-22 三菱化学株式会社 薬剤含有タンパク質結合リポソーム
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
WO1993001286A2 (en) 1991-06-28 1993-01-21 Massachusetts Institute Of Technology Localized oligonucleotide therapy
NZ244306A (en) 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
US5521291A (en) 1991-09-30 1996-05-28 Boehringer Ingelheim International, Gmbh Conjugates for introducing nucleic acid into higher eucaryotic cells
FR2692265B1 (fr) 1992-05-25 1996-11-08 Centre Nat Rech Scient Composes biologiquement actifs de type phosphotriesters.
EP1498427B1 (en) 1992-08-21 2009-12-16 Vrije Universiteit Brussel Immunoglobulins devoid of light chains
US6005079A (en) 1992-08-21 1999-12-21 Vrije Universiteit Brussels Immunoglobulins devoid of light chains
US5583020A (en) 1992-11-24 1996-12-10 Ribozyme Pharmaceuticals, Inc. Permeability enhancers for negatively charged polynucleotides
JP3351476B2 (ja) 1993-01-22 2002-11-25 三菱化学株式会社 リン脂質誘導体及びそれを含有するリポソーム
US5395619A (en) 1993-03-03 1995-03-07 Liposome Technology, Inc. Lipid-polymer conjugates and liposomes
ES2201076T3 (es) 1993-03-19 2004-03-16 The Johns Hopkins University School Of Medicine Factor-8 de diferenciacion del crecimiento.
US20030074680A1 (en) 1993-03-19 2003-04-17 Johns Hopkins University School Of Medicine Growth differentiation factor-8
US5994618A (en) 1997-02-05 1999-11-30 Johns Hopkins University School Of Medicine Growth differentiation factor-8 transgenic mice
US5462854A (en) 1993-04-19 1995-10-31 Beckman Instruments, Inc. Inverse linkage oligonucleotides for chemical and enzymatic processes
DK0698097T3 (da) 1993-04-29 2001-10-08 Unilever Nv Produktion af antistoffer eller (funktionaliserede) fragmenter deraf afledt af Camelidae-immunoglobuliner med tung kæde
DE69435005T2 (de) 1993-05-11 2008-04-17 The University Of North Carolina At Chapel Hill Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
US5534259A (en) 1993-07-08 1996-07-09 Liposome Technology, Inc. Polymer compound and coated particle composition
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5417978A (en) 1993-07-29 1995-05-23 Board Of Regents, The University Of Texas System Liposomal antisense methyl phosphonate oligonucleotides and methods for their preparation and use
US6060456A (en) 1993-11-16 2000-05-09 Genta Incorporated Chimeric oligonucleoside compounds
US5595756A (en) 1993-12-22 1997-01-21 Inex Pharmaceuticals Corporation Liposomal compositions for enhanced retention of bioactive agents
US5543152A (en) 1994-06-20 1996-08-06 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5591721A (en) 1994-10-25 1997-01-07 Hybridon, Inc. Method of down-regulating gene expression
US5512295A (en) 1994-11-10 1996-04-30 The Board Of Trustees Of The Leland Stanford Junior University Synthetic liposomes for enhanced uptake and delivery
US5707618A (en) 1995-03-24 1998-01-13 Genzyme Corporation Adenovirus vectors for gene therapy
TW311927B (enExample) 1995-07-11 1997-08-01 Minnesota Mining & Mfg
US7803782B2 (en) 2003-05-28 2010-09-28 Roche Madison Inc. Intravenous delivery of polynucleotides to cells in mammalian limb
DK2111876T3 (da) 1995-12-18 2011-12-12 Angiodevice Internat Gmbh Tværbundne polymerpræparater og fremgangsmåder til anvendelse deraf
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
WO1997040854A2 (en) 1996-05-01 1997-11-06 Antivirals Inc. Polypeptide conjugates for transporting substances across cell membranes
IL132941A0 (en) 1997-05-21 2001-03-19 Univ Leland Stanford Junior Composition and method for enhancing transport across biological membranes
US6103466A (en) 1997-07-14 2000-08-15 University Of Liege Double-muscling in mammals
WO1999002667A1 (en) 1997-07-14 1999-01-21 University Of Liege Mutations in the myostation gene cause double-muscling in mammals
US6133246A (en) 1997-08-13 2000-10-17 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the modulation of JNK proteins
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6228579B1 (en) 1997-11-14 2001-05-08 San Diego State University Foundation Method for identifying microbial proliferation genes
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
US7094765B1 (en) 1999-01-29 2006-08-22 Avi Biopharma, Inc. Antisense restenosis composition and method
KR20010102992A (ko) 1999-01-29 2001-11-17 추후보정 표적 ｒｎａ를 검출하는 비-침입적 방법
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6284882B1 (en) 1999-06-10 2001-09-04 Abbott Laboratories Myostatin gene promoter and inhibition of activation thereof
AU4889100A (en) 1999-07-30 2001-02-01 Pfizer Products Inc. Myostatin regulatory region, nucleotide sequence determination and methods for its use
US6669951B2 (en) 1999-08-24 2003-12-30 Cellgate, Inc. Compositions and methods for enhancing drug delivery across and into epithelial tissues
US6677153B2 (en) 1999-11-29 2004-01-13 Avi Biopharma, Inc. Antisense antibacterial method and composition
KR20020079768A (ko) 2000-01-04 2002-10-19 에이브이아이 바이오파마 인코포레이티드 안티센스 항박테리아 세포분열 조성물 및 방법
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
JP2004509604A (ja) 2000-03-28 2004-04-02 アイシス・ファーマシューティカルス・インコーポレーテッド ｍＲＮＡプロセッシングのアンチセンスモジュレーションによる、細胞行動の改変
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
EP3020804A1 (en) 2000-07-06 2016-05-18 Sarepta Therapeutics, Inc. Transforming growth factor beta (tgf- ) blocking agent-treated stem cell composition and method
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US20050124566A1 (en) 2001-05-18 2005-06-09 Sirna Therapeutics, Inc. RNA interference mediated inhibition of myostatin gene expression using short interfering nucleic acid (siNA)
CA2459347C (en) 2001-09-04 2012-10-09 Exiqon A/S Locked nucleic acid (lna) compositions and uses thereof
CA2463641C (en) 2001-10-16 2013-07-09 Avi Biopharma, Inc. Antisense antiviral agent and method for treating ssrna viral infection
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
JP2003203451A (ja) 2001-12-28 2003-07-18 Fujitsu Ltd ヘッドスライダ
KR100464261B1 (ko) 2002-01-24 2005-01-03 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US20090099117A1 (en) 2002-02-20 2009-04-16 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF MYOSTATIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
KR20030084444A (ko) 2002-04-26 2003-11-01 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
WO2004044140A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceticals, Inc. 2’-substituted oligomeric compounds and compositions for use in gene modulations
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
ES2351976T3 (es) 2003-04-29 2011-02-14 Avi Biopharma, Inc. Composiciones para mejorar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células.
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
EP1713332A4 (en) 2004-01-23 2010-08-18 Avi Biopharma Inc ANTISENSE OLIGOMERS AND METHOD FOR INDUCTION OF IMMUNOTHERAPY AND IMMUNOSUPPRESSION
EP1729793B1 (en) 2004-03-02 2015-07-22 Acceleron Pharma Inc. Alk7 and myostatin inhibitors for muscle wasting disorders
WO2005107447A2 (en) 2004-05-11 2005-11-17 Ingenium Pharmaceuticals Ag Methods for the production of improved live stocks and disease models for therapeutic research
SI2206781T1 (sl) 2004-06-28 2016-05-31 The University Of Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe
NZ538097A (en) 2005-02-07 2006-07-28 Ovita Ltd Method and compositions for improving wound healing
ES2852549T3 (es) * 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
AU2015203791B2 (en) 2005-02-09 2017-06-29 Sarepta Therapeutics, Inc. Antisense composition and method for treating
CA2605512A1 (en) 2005-04-22 2006-10-26 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secondary rna structure.
BRPI0610248A2 (pt) 2005-04-25 2010-06-08 Pfizer anticorpos contra miostatina, composição farmacêutica compreendendo os mesmos, linhas celulares que os produzem , moléculas de ácido nucleico codificantes dos mesmos bem como seus usos
CA2538208A1 (en) 2005-05-04 2006-11-04 Universite Laval Modulation of myostatin and use thereof in cell transplantation-based treatment of muscle disease
US8361977B2 (en) 2005-06-23 2013-01-29 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of SMN2 splicing
WO2007003216A1 (en) 2005-07-06 2007-01-11 Universidad Autónoma de Madrid Anti-ccr7 receptor antibodies for the treatment of cancer
US8501704B2 (en) 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
US7785834B2 (en) 2005-11-10 2010-08-31 Ercole Biotech, Inc. Soluble TNF receptors and their use in treatment of disease
US20070105807A1 (en) 2005-11-10 2007-05-10 Sazani Peter L Splice switch oligomers for TNF superfamily receptors and their use in treatment of disease
EP2089521A1 (en) 2006-02-08 2009-08-19 Ercole Biotech, Inc. Soluble tnf receptors and their use in treatment of disease
ES2657400T3 (es) * 2006-05-10 2018-03-05 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
US8097596B2 (en) 2006-06-30 2012-01-17 Lakewood-Amedex, Inc. Compositions and methods for the treatment of muscle wasting
WO2008005019A1 (en) * 2006-07-07 2008-01-10 Dale Roderic M K Compositions and methods for the treatment of muscle wasting
US20090264353A1 (en) 2007-10-19 2009-10-22 Santaris Pharma A/S Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease
TW201940502A (zh) 2007-02-02 2019-10-16 美商艾瑟勒朗法瑪公司 衍生自ＡｃｔＲＩＩＢ的變體與其用途
KR20170056032A (ko) 2007-05-01 2017-05-22 로슈 이노베이션 센터 코펜하겐 에이/에스 Ｔｎｆ 슈퍼패밀리 수용체에 대한 스플라이스 스위칭 올리고머 및 질병 치료에 있어서의 그의 용도
EP2173373B1 (en) 2007-06-06 2020-04-15 Sarepta Therapeutics, Inc. Soluble her2 and her3 splice variant proteins, splice-switching oligonucleotides, and their use in the treatment of disease
AU2008271050B2 (en) * 2007-06-29 2014-11-06 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2009008725A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
US7935816B2 (en) 2007-10-25 2011-05-03 Gene Tools, Llc Molecular transporter compositions comprising dendrimeric oligoguanidine with a triazine core that facilitate delivery into cells in vivo
DK2203173T3 (en) 2007-10-26 2016-02-29 Academisch Ziekenhuis Leiden Materials and methods for prevention of muscle diseases
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
CA2884340C (en) 2007-11-15 2017-07-25 Sarepta Therapeutics, Inc. Method of synthesis of morpholino oligomers
CA2710013A1 (en) 2007-12-28 2009-07-09 Avi Biopharma, Inc. Immunomodulatory agents and methods of use
CA2711302C (en) 2008-01-04 2016-11-08 Cue Acoustics, Inc. Audio device with integrated switching power supply
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
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
LT3750552T (lt) 2008-08-14 2023-06-26 Acceleron Pharma Inc. Gdf gaudyklės
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2762567B1 (en) 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
GB0821457D0 (en) * 2008-11-24 2008-12-31 Trillion Genomics Ltd Oligonucleotides
US8592386B2 (en) 2008-12-17 2013-11-26 Sarepta Therapeutics, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
CN102625840A (zh) 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
WO2010120820A1 (en) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
ES2593836T3 (es) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la DMD
EA027071B1 (ru) 2009-04-27 2017-06-30 Новартис Аг АНТИТЕЛО К ActRIIB И СОДЕРЖАЩАЯ ЕГО КОМПОЗИЦИЯ
WO2010129406A2 (en) 2009-05-04 2010-11-11 The Johns Hopkins University Methods of promoting muscle growth
PT3449926T (pt) 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
SMT201800579T1 (it) 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
JP2013530154A (ja) 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
PL2623507T3 (pl) 2010-09-30 2017-03-31 Nippon Shinyaku Co., Ltd. Pochodna kwasu morfolinonukleinowego
EP2672977A1 (en) 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
WO2012150960A1 (en) * 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
US20130085139A1 (en) * 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
ES2535654T3 (es) 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
BR112014011875B1 (pt) 2011-11-18 2022-01-04 Sarepta Therapeutics, Inc Oligonucleotídeos funcionalmente modificados e subunidades dos mesmos
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
CN108611349A (zh) 2011-12-28 2018-10-02 日本新药株式会社 反义核酸
ES2907250T3 (es) 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
AU2013285698A1 (en) 2012-07-03 2015-02-19 Biomarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
KR20150099804A (ko) 2012-12-20 2015-09-01 사렙타 쎄러퓨틱스 인코퍼레이티드 근위축증을 치료하기 위한 개선된 엑손 스키핑 조성물
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
NZ732507A (en) 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
WO2014172448A2 (en) 2013-04-17 2014-10-23 Anaptysbio, Inc. Antibodies directed against activin receptor type ii (actrii)
IL282239B2 (en) * 2013-09-05 2023-10-01 Sarepta Therapeutics Inc Antisense-induced exon2 inclusion in acid alpha-glucosidase
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2017062835A2 (en) 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
JP2021026584A (ja) 2019-08-07 2021-02-22 ｉ Ｓｍａｒｔ Ｔｅｃｈｎｏｌｏｇｉｅｓ株式会社 改善対策リコメンドシステム

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