HK1203835A1 - Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy rna - Google Patents

Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy rna

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Publication number
HK1203835A1
HK1203835A1 HK15104532.0A HK15104532A HK1203835A1 HK 1203835 A1 HK1203835 A1 HK 1203835A1 HK 15104532 A HK15104532 A HK 15104532A HK 1203835 A1 HK1203835 A1 HK 1203835A1
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HK
Hong Kong
Prior art keywords
rna
duchenne
treatment
muscular dystrophy
improved characteristics
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HK15104532.0A
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English (en)
Chinese (zh)
Inventor
Peter Christian De Visser
Judith Christina Theodora Van Deutekom
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Biomarin Technologies B V
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=48873711&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HK1203835(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Biomarin Technologies B V filed Critical Biomarin Technologies B V
Publication of HK1203835A1 publication Critical patent/HK1203835A1/xx

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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
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    • C12N2320/33Alteration of splicing

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HK15104532.0A 2012-01-27 2015-05-13 Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy rna HK1203835A1 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201261591354P 2012-01-27 2012-01-27
EP12152934 2012-01-27
US201261612467P 2012-03-19 2012-03-19
PCT/NL2013/050045 WO2013112053A1 (en) 2012-01-27 2013-01-28 Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy

Publications (1)

Publication Number Publication Date
HK1203835A1 true HK1203835A1 (en) 2015-11-06

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HK15104532.0A HK1203835A1 (en) 2012-01-27 2015-05-13 Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy rna

Country Status (12)

Country Link
US (4) US20150045413A1 (pt)
EP (2) EP2806900B1 (pt)
JP (4) JP2015509922A (pt)
CN (3) CN111893117B (pt)
AU (3) AU2013212758A1 (pt)
BR (1) BR112014018427B1 (pt)
CA (2) CA3120918A1 (pt)
ES (1) ES2907250T3 (pt)
HK (1) HK1203835A1 (pt)
IL (1) IL233809B (pt)
NZ (1) NZ627896A (pt)
WO (1) WO2013112053A1 (pt)

Families Citing this family (83)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
EP3228711A1 (en) 2004-06-28 2017-10-11 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
WO2008018795A1 (en) 2006-08-11 2008-02-14 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
PT2203173E (pt) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
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
CN105779453A (zh) 2008-10-24 2016-07-20 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
JP5963678B2 (ja) 2009-11-12 2016-08-03 ジ ユニバーシティ オブ ウェスタン オーストラリア 病状を治療するためのアンチセンス分子及び方法
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CA3120918A1 (en) 2012-01-27 2013-08-01 Biomarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
BR112014033004B1 (pt) * 2012-07-03 2021-10-19 Biomarin Technologies B.V. Oligonucleotídeo para tratamento de pacientes com distrofia muscular
US20160040162A1 (en) * 2013-03-14 2016-02-11 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
JP6449231B2 (ja) 2013-03-14 2019-01-09 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィを処置するためのエキソンスキッピング組成物
MX2015013117A (es) 2013-03-15 2016-07-14 Sarepta Therapeutics Inc Composiciones mejoradas para tratar distrofia muscular.
EP4389903A2 (en) 2013-06-05 2024-06-26 Duke University Rna-guided gene editing and gene regulation
WO2015035231A1 (en) 2013-09-05 2015-03-12 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
US10059947B2 (en) * 2013-09-11 2018-08-28 Synthena Ag Nucleic acids and methods for the treatment of Pompe disease
SG10201912858VA (en) 2014-06-17 2020-02-27 Nippon Shinyaku Co Ltd Antisense nucleic acids
US10517898B2 (en) 2014-11-20 2019-12-31 The Regents Of The University Of California Compositions and methods related to hematologic recovery
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
EP3302497A4 (en) 2015-06-01 2019-01-16 Sarepta Therapeutics, Inc. EXCLUSION OF EXON INDUCED PAT TECHNOLOGY ANTISENSE IN COLLAGEN TYPE VII
MA43072A (fr) * 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
MX2018002339A (es) 2015-08-25 2018-12-19 Univ Duke Composiciones y metodos de mejora de la especificidad en ingenieria genomica usando endonucleasas guiadas por arn.
EP3778895A1 (en) 2015-09-15 2021-02-17 Nippon Shinyaku Co., Ltd. Antisense nucleic acid
MA45819A (fr) * 2015-10-09 2018-08-15 Sarepta Therapeutics Inc Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés
KR20180056766A (ko) * 2015-10-09 2018-05-29 웨이브 라이프 사이언시스 리미티드 뉴클레오티드 조성물 및 이의 방법
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
WO2017134525A1 (en) 2016-02-02 2017-08-10 Olix Pharmaceuticals, Inc. TREATMENT OF ATOPIC DERMATITIS AND ASTHMA USING RNA COMPLEXES THAT TARGET lL4Rα, TRPA1, OR F2RL1
EP3445405A4 (en) 2016-04-18 2019-12-18 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMERS AND METHOD FOR USE THEREOF TO TREAT DISEASES RELATING TO THE ACID ALPHA GLUCOSIDASE GENE
EA201892467A1 (ru) 2016-04-29 2019-05-31 Сарепта Терапьютикс, Инк. Олигонуклеотидные аналоги, нацеленные на lmna человека
CN109311919A (zh) 2016-06-30 2019-02-05 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体
IL301091A (en) * 2016-07-05 2023-05-01 Biomarin Tech Bv Oligonucleotides containing a bicyclic skeleton and their use for the treatment of genetic diseases
WO2018017814A1 (en) 2016-07-20 2018-01-25 President And Fellows Of Harvard College Peptidoglycan glycosyltransferase inhibitors of sed proteins for treating bacterial infections
WO2018053142A2 (en) 2016-09-14 2018-03-22 President And Fellows Of Harvard College Methods and compositions for modulating erythropoiesis
MX2019005671A (es) 2016-11-16 2020-07-20 Academisch Ziekenhuis Leiden Sustancias para direccionar diversos organos o tejidos seleccionados.
WO2018112033A1 (en) 2016-12-13 2018-06-21 President And Fellows Of Harvard College Methods and compositions for targeting tumor-infiltrating tregs
CN110290812B (zh) 2016-12-19 2023-06-02 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物
DK4122497T3 (da) 2016-12-19 2024-06-10 Sarepta Therapeutics Inc Exonskipping-oligomerkonjugater til muskeldystrofi
DK3554552T3 (da) 2016-12-19 2022-10-24 Sarepta Therapeutics Inc Exon-skipping-oligomerkonjugater mod muskeldystrofi
AU2018256435A1 (en) 2017-04-20 2019-11-07 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
AU2018337854A1 (en) * 2017-09-22 2020-03-19 Katarzyna JASTRZEBSKA Thiomorpholino oligonucleotides for the treatment of muscular dystrophy
JP2020536058A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを処置するための併用療法
JP2020536060A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを処置するための併用療法
US20200254002A1 (en) 2017-09-28 2020-08-13 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
JP7394753B2 (ja) * 2017-10-18 2023-12-08 サレプタ セラピューティクス, インコーポレイテッド アンチセンスオリゴマー化合物
JPWO2019176864A1 (ja) * 2018-03-12 2021-02-25 国立大学法人 岡山大学 筋ジストロフィー関連心筋症の治療又は予防剤
EP3790596A4 (en) * 2018-05-11 2022-04-06 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US20220251551A1 (en) * 2018-06-13 2022-08-11 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
EP4219717A3 (en) 2018-06-13 2023-12-20 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
EP3815696A4 (en) * 2018-06-26 2022-11-30 Nippon Shinyaku Co., Ltd. COMPOSITION COMPRISING AN ANTISENSE OLIGONUCLEOTIDE AND ITS USE FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
TW202020153A (zh) 2018-07-27 2020-06-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
US11273137B2 (en) 2018-09-04 2022-03-15 Board Of Trustees Of Michigan State University Methods and compositions to prevent and treat disorders associated with mutations in the ODC1 gene
EA202191601A1 (ru) 2018-12-13 2022-01-19 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
CN114949201A (zh) 2019-03-12 2022-08-30 哈佛大学校长及研究员协会 治疗癌症的方法和组合物
WO2020214763A1 (en) 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
WO2020252257A1 (en) 2019-06-12 2020-12-17 President And Fellows Of Harvard College Methods and compositions for modulation of an interspecies gut bacterial pathway for levodopa metabolism
KR102216561B1 (ko) * 2019-09-06 2021-02-17 서울대학교산학협력단 Roquin을 표적으로 하는 사이토메갈로바이러스 감염 억제제 스크리닝 방법
IL293997A (en) * 2019-12-19 2022-08-01 Nippon Shinyaku Co Ltd Antistrand nucleic acids that allow exon skipping
IL295967A (en) * 2020-02-28 2022-10-01 Nippon Shinyaku Co Ltd Antisense nucleic acids for splicing on exon 51
WO2021178556A1 (en) 2020-03-04 2021-09-10 Regeneron Pharmaceuticals, Inc. Methods and compositions for sensitization of tumor cells to immune therapy
EP4157264A4 (en) 2020-05-27 2024-06-19 Univ California COMPOSITIONS AND METHODS FOR TRANSDIFFERENTIATION OF CELLS
CN115011598A (zh) * 2020-09-02 2022-09-06 西湖大学 杜氏肌营养不良症相关的外显子剪接增强子、sgRNA、基因编辑工具及应用
WO2022056454A2 (en) 2020-09-14 2022-03-17 President And Fellows Of Harvard College Methods and compositions for treating hpv-positive cancers
US20230340124A1 (en) 2020-09-16 2023-10-26 President And Fellows Of Harvard College Methods of treating an individual that has failed an anti-pd-1/anti-pd-l1 therapy
WO2022217366A1 (en) * 2021-04-14 2022-10-20 The Royal Institution For The Advancement Of Learning/Mcgill University Modified oligonucleotides for the treatment of duchenne muscular dystrophy
EP4330393A1 (en) 2021-04-27 2024-03-06 Research Institute at Nationwide Children's Hospital Products and methods for inducing exon 2 skipping of the dmd gene in treating muscular dystrophy
WO2023055774A1 (en) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
WO2023070086A1 (en) 2021-10-22 2023-04-27 Sarepta Therapeutics, Inc. Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
WO2023091704A1 (en) 2021-11-18 2023-05-25 Circularis Biotechnologies, Inc. Compositions and methods for production of circular nucleic acid molecules
WO2023096847A2 (en) 2021-11-24 2023-06-01 Prime Medicine, Inc. Methods and compositions for inhibiting mismatch repair
WO2023150181A1 (en) 2022-02-01 2023-08-10 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
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
US20240182561A1 (en) 2022-11-04 2024-06-06 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes

Family Cites Families (219)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5541308A (en) 1986-11-24 1996-07-30 Gen-Probe Incorporated Nucleic acid probes for detection and/or quantitation of non-viral organisms
US5766847A (en) 1988-10-11 1998-06-16 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Process for analyzing length polymorphisms in DNA regions
DE3834636A1 (de) 1988-10-11 1990-04-19 Max Planck Gesellschaft Verfahren zur analyse von laengenpolymorphismen in dna-bereichen
US6867195B1 (en) 1989-03-21 2005-03-15 Vical Incorporated Lipid-mediated polynucleotide administration to reduce likelihood of subject's becoming infected
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
FR2675803B1 (fr) 1991-04-25 1996-09-06 Genset Sa Oligonucleotides fermes, antisens et sens et leurs applications.
CA2082411A1 (en) 1991-06-28 1992-12-29 Robert D. Rosenberg Localized oligonucleotide therapy
JPH07501204A (ja) 1991-06-28 1995-02-09 マサチューセッツ インスティテュート オブ テクノロジー 局所的オリゴヌクレオチド療法
US6200747B1 (en) 1992-01-28 2001-03-13 North Shore University Hospital Research Corp. Method and kits for detection of fragile X specific, GC-rich DNA sequences
US5869252A (en) 1992-03-31 1999-02-09 Abbott Laboratories Method of multiplex ligase chain reaction
US6172208B1 (en) 1992-07-06 2001-01-09 Genzyme Corporation Oligonucleotides modified with conjugate groups
US5418139A (en) 1993-02-10 1995-05-23 University Of Iowa Research Foundation Method for screening for cardiomyopathy
CA2116280A1 (en) 1993-03-05 1994-09-06 Marcy E. Macdonald Huntingtin dna, protein and uses thereof
CA2162361C (en) 1993-05-11 2008-10-21 Ryszard Kole Antisense oligonucleotides which combat aberrant splicing and methods of using the same
US5695933A (en) 1993-05-28 1997-12-09 Massachusetts Institute Of Technology Direct detection of expanded nucleotide repeats in the human genome
US5741645A (en) 1993-06-29 1998-04-21 Regents Of The University Of Minnesota Gene sequence for spinocerebellar ataxia type 1 and method for diagnosis
US5624803A (en) 1993-10-14 1997-04-29 The Regents Of The University Of California In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides derived therefrom
US5627263A (en) 1993-11-24 1997-05-06 La Jolla Cancer Research Foundation Integrin-binding peptides
DE4342605A1 (de) 1993-12-14 1995-06-22 Buna Gmbh Funktionalisierte Olefinhomo- und -copolymere
US5962332A (en) 1994-03-17 1999-10-05 University Of Massachusetts Detection of trinucleotide repeats by in situ hybridization
EP0758403B1 (en) 1994-05-05 1998-06-24 Beckman Instruments, Inc. Oligonucleotide repeat arrays
US5968909A (en) 1995-08-04 1999-10-19 Hybridon, Inc. Method of modulating gene expression with reduced immunostimulatory response
US5854223A (en) 1995-10-06 1998-12-29 The Trustees Of Columbia University In The City Of New York S-DC28 as an antirestenosis agent after balloon injury
US20070173465A9 (en) 1995-10-11 2007-07-26 Monahan Sean D Expression of zeta negative and zeta positive nucleic acids using a dystrophin gene
US7034009B2 (en) 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US6300060B1 (en) 1995-11-09 2001-10-09 Dana-Farber Cancer Institute, Inc. Method for predicting the risk of prostate cancer morbidity and mortality
DE69729179T2 (de) 1996-02-14 2004-12-30 Isis Pharmaceuticals, Inc., Carlsbad Lückenhaft Zucker-modifizierte Oligonukleotide
JP3950480B2 (ja) 1996-07-18 2007-08-01 株式会社エスアールエル 脊髄小脳変性症2型の原因遺伝子の検出方法及びそのためのプライマー
CA2241173A1 (en) 1996-10-30 1998-05-07 Srl, Inc. Cdna fragment of causative gene of spinocerebellar ataxia type 2
US5853995A (en) 1997-01-07 1998-12-29 Research Development Foundation Large scale genotyping of diseases and a diagnostic test for spinocerebellar ataxia type 6
WO1998043993A2 (en) 1997-03-31 1998-10-08 Yale University Nucleic acid catalysts
US20020137890A1 (en) 1997-03-31 2002-09-26 Genentech, Inc. Secreted and transmembrane polypeptides and nucleic acids encoding the same
WO1998049345A1 (en) 1997-04-29 1998-11-05 Trustees Of Boston University Methods and compositions for targeted dna differential display
US6329501B1 (en) 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
US6514755B1 (en) 1998-08-18 2003-02-04 Regents Of The University Of Minnesota SCA7 gene and methods of use
US6280938B1 (en) 1997-08-19 2001-08-28 Regents Of The University Of Minnesota SCA7 gene and method of use
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
EP1030913A2 (en) 1997-09-22 2000-08-30 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Nucleic acid catalysts with endonuclease activity
US6130207A (en) 1997-11-05 2000-10-10 South Alabama Medical Science Foundation Cell-specific molecule and method for importing DNA into a nucleus
JP3012923B2 (ja) 1998-01-26 2000-02-28 新潟大学長 Cagリピート病の治療薬
KR100280219B1 (ko) 1998-02-26 2001-04-02 이수빈 삼핵산 반복 서열을 이용한 신경정신 질환의 진단 방법 및 진단 시약
US6683173B2 (en) 1998-04-03 2004-01-27 Epoch Biosciences, Inc. Tm leveling methods
US6322978B1 (en) 1998-04-20 2001-11-27 Joslin Diabetes Center, Inc. Repeat polymorphism in the frataxin gene and uses therefore
CA2330574A1 (en) 1998-04-29 1999-11-04 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into ribozymes
DE69919869T2 (de) 1998-06-10 2005-09-29 Biognostik Gesellschaft für Biomolekulare Diagnostik mbH Stimulierung des immunsystems
US6924355B2 (en) 1998-09-01 2005-08-02 Genentech, Inc. PRO1343 polypeptides
EP1115847A1 (en) 1998-09-25 2001-07-18 Children's Medical Center Corporation Short peptides which selectively modulate the activity of protein kinases
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6365577B1 (en) 1998-10-26 2002-04-02 Avi Biopharma, Inc. p53 antisense agent and method
US6399575B1 (en) 1998-11-10 2002-06-04 Auburn University Methods and compositions for targeting compounds to the central nervous system
US6133031A (en) 1999-08-19 2000-10-17 Isis Pharmaceuticals Inc. Antisense inhibition of focal adhesion kinase expression
US20040226056A1 (en) 1998-12-22 2004-11-11 Myriad Genetics, Incorporated Compositions and methods for treating neurological disorders and diseases
US20020049173A1 (en) 1999-03-26 2002-04-25 Bennett C. Frank Alteration of cellular behavior by antisense modulation of mRNA processing
US6379698B1 (en) 1999-04-06 2002-04-30 Isis Pharmaceuticals, Inc. Fusogenic lipids and vesicles
IL145586A0 (en) 1999-04-08 2002-06-30 Oasis Biosciences Inc Antisense oligonucleotides comprising universal and/or degenerate bases
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
US20030236214A1 (en) 1999-06-09 2003-12-25 Wolff Jon A. Charge reversal of polyion complexes and treatment of peripheral occlusive disease
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
WO2000078813A2 (en) 1999-06-18 2000-12-28 Emory University Huntington disease cellular model: stably transfected pc12 cells expressing mutant huntingtin
CA2403243A1 (en) 1999-08-31 2001-03-08 Ribozyme Pharmaceuticals, Inc. Nucleic acid based modulators of gene expression
CA2386239A1 (en) 1999-10-04 2001-04-12 University Of Medicine And Dentistry Of New Jersey Methods for identifying rna binding compounds
US6165786A (en) 1999-11-03 2000-12-26 Isis Pharmaceuticals, Inc. Antisense modulation of nucleolin expression
WO2001059102A2 (en) 2000-02-08 2001-08-16 Ribozyme Pharmaceuticals, Inc. Nucleozymes with endonuclease activity
CN1311202A (zh) * 2000-02-29 2001-09-05 复旦大学 一种新的多肽——人反转录相关因子22和编码这种多肽的多核苷酸
EP1133993A1 (en) 2000-03-10 2001-09-19 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Substances for the treatment of spinal muscular atrophy
AU4819101A (en) 2000-04-13 2001-10-30 University Of British Columbia, The Modulating cell survival by modulating huntingtin function
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
AU2001261063A1 (en) 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
ATE292638T1 (de) 2000-05-01 2005-04-15 Hybridon Inc Modulation von oligonukleotid cpg-vermittelter immunstimulation durch modifikation der nucleoside
WO2001085751A1 (en) 2000-05-09 2001-11-15 Reliable Biopharmaceutical, Inc. Polymeric compounds useful as prodrugs
CA2410758A1 (en) 2000-05-31 2001-12-06 Genset S.A. Use of acrp30 globular head to promote increases in muscle mass and muscle differentiation
CN1326990A (zh) 2000-06-07 2001-12-19 上海博德基因开发有限公司 一种新的多肽——人类dna cgg重复结合蛋白16.17和编码这种多肽的多核苷酸
US20030124523A1 (en) 2000-06-22 2003-07-03 Asselbergs Fredericus Alphonsus Maria Organic compounds
US6794192B2 (en) 2000-06-29 2004-09-21 Pfizer Inc. Target
RU2165149C1 (ru) 2000-07-03 2001-04-20 Шапошников Валерий Геннадьевич Система формования и упаковки изделий из сахарной ваты
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
JP4836366B2 (ja) * 2000-08-25 2011-12-14 雅文 松尾 デュシェンヌ型筋ジストロフィー治療剤
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
AU3922802A (en) 2000-10-02 2002-05-27 Keck Graduate Inst Methods for identifying nucleotides at defined positions in target nucleic acidsusing fluorescence polarization
EP1366160B1 (en) 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
US6623927B1 (en) 2000-11-08 2003-09-23 Council Of Scientific And Industrial Research Method of detection of allelic variants of SCA2 gene
AU2002236499A8 (en) 2000-11-30 2009-12-03 Uab Research Foundation Receptor-mediated uptake of peptides that bind the human transferrin receptor
US7001994B2 (en) 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
TWI329129B (en) 2001-02-08 2010-08-21 Wyeth Corp Modified and stabilized gdf propeptides and uses thereof
KR100408053B1 (ko) 2001-02-13 2003-12-01 엘지전자 주식회사 에스알엠의 토크리플 저감방법
AU2002256359A1 (en) 2001-04-24 2002-11-05 Epigenesis Pharmaceuticals, Inc. Antisense and anti-inflammatory based compositions to treat respiratory disorders
WO2002092763A2 (en) 2001-05-11 2002-11-21 Regents Of The University Of Minnesota Intron associated with myotonic dystrophy type 2 and methods of use
US20050277133A1 (en) 2001-05-18 2005-12-15 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US20050014172A1 (en) 2002-02-20 2005-01-20 Ivan Richards RNA interference mediated inhibition of muscarinic cholinergic receptor gene expression using short interfering nucleic acid (siNA)
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
EP1627061B1 (en) 2001-05-18 2009-08-12 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
IL143379A (en) 2001-05-24 2013-11-28 Yissum Res Dev Co Oligonucleotide against human ache isoform r and its uses
AU2002316421B2 (en) 2001-06-26 2008-05-15 Bristol-Myers Squibb Company N-heterocyclic inhibitors of TNF-ALPHA expression
JP4903984B2 (ja) 2001-07-06 2012-03-28 トピジェン・ファーマシューティカルズ・インコーポレーテッド オリゴヌクレオチドのinvivoでの効率を増加するおよび哺乳動物において炎症を阻害するための方法
AU2002326589B2 (en) 2001-08-07 2008-06-05 University Of Delaware Compositions and methods for the prevention and treatment of Huntington's disease
CA2455336A1 (en) 2001-08-10 2003-02-20 Novartis Ag Peptides that bind to atherosclerotic lesions
US20060074034A1 (en) 2001-09-17 2006-04-06 Collins Douglas A Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
KR20030035047A (ko) 2001-10-29 2003-05-09 (주)바이오코돈 Bmp-4 유전자 발현을 이용한 편평태선 질환의 치료 및진단방법
AU2002363253A1 (en) 2001-11-01 2003-05-12 Gpc Biotech Inc. Endothelial-cell binding peptides for diagnosis and therapy
US20030134790A1 (en) 2002-01-11 2003-07-17 University Of Medicine And Dentistry Of New Jersey Bone Morphogenetic Protein-2 And Bone Morphogenetic Protein-4 In The Treatment And Diagnosis Of Cancer
US20030203356A1 (en) 2002-01-22 2003-10-30 The Cleveland Clinic Foundation RNase L activator-antisense complexes
WO2003069330A1 (en) 2002-02-11 2003-08-21 The Trustees Of Columbia University In The City Of New York System and method for identifying proteins involved in force-initiated signal transduction
US20050096284A1 (en) 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
DK1487493T3 (da) 2002-03-01 2010-05-25 Univ Tulane Konjugater af cytotoksiske midler og biologisk aktive peptider
US20040101852A1 (en) 2002-11-21 2004-05-27 Isis Pharmaceuticals Inc. Modulation of CGG triplet repeat binding protein 1 expression
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
US20040102395A1 (en) 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of IAP-like expression
EP1380644A1 (en) 2002-07-08 2004-01-14 Kylix B.V. The use of specified TCF target genes to identify drugs for the treatment of cancer, in particular colorectal cancer, in which TCF/beta-catenin/WNT signalling plays a central role
EP1585560A4 (en) 2002-07-26 2011-03-16 Mirus Bio Corp ADMINISTRATION OF MOLECULES AND COMPLEXES TO IN VIVO MAMMAL CELLS
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
JP2005535318A (ja) 2002-08-12 2005-11-24 ユニヴェルシテ ドゥ シェルブルック プレメッセンジャーrnaのスプライス部位選択の再プログラミング方法
GB0219143D0 (en) 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
US20040219565A1 (en) * 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
DE60219215T2 (de) 2002-10-23 2008-01-03 Centre For Research And Technology Hellas/Intitute Of Agrobiotechnology In.A, Thermi Prionen-bindende peptidsequenzen
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7655785B1 (en) 2002-11-14 2010-02-02 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
JP4777777B2 (ja) 2002-11-25 2011-09-21 雅文 松尾 mRNA前駆体のスプライシングを修飾するENA核酸医薬
GB0228079D0 (en) 2002-12-02 2003-01-08 Laxdale Ltd Huntington's Disease
EP2216407B1 (en) 2003-03-07 2016-01-13 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
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
US7514551B2 (en) 2003-04-03 2009-04-07 Enzo Life Sciences, Inc. Multisignal labeling reagents, and processes and uses therefor
CA2526893C (en) 2003-05-14 2010-10-26 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
AU2004245025A1 (en) 2003-06-02 2004-12-16 Wyeth Use of myostatin (GDF8) inhibitors in conjunction with corticosteroids for treating neuromuscular disorders
DK1495769T3 (da) 2003-07-11 2008-06-23 Lbr Medbiotech B V Mannose-6-phosphat-receptormedieret gentransfer til muskelceller
US20050048495A1 (en) 2003-08-29 2005-03-03 Baker Brenda F. Isoform-specific targeting of splice variants
US20050054752A1 (en) 2003-09-08 2005-03-10 O'brien John P. Peptide-based diblock and triblock dispersants and diblock polymers
WO2005033134A2 (en) 2003-09-30 2005-04-14 Regeneron Pharmaceuticals, Inc. Secreted protein therapeutics and uses thereof
JP2007508030A (ja) 2003-10-14 2007-04-05 カーネル・バイオファーマ・インコーポレイテッド 血液脳関門を介してpnaを送達するための2相pna結合体
US20050191636A1 (en) 2004-03-01 2005-09-01 Biocept, Inc. Detection of STRP, such as fragile X syndrome
US20080207538A1 (en) 2004-03-11 2008-08-28 Lawrence David S Enhanced Production of Functional Proteins From Defective Genes
WO2005105995A2 (en) 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2005116204A1 (ja) 2004-05-11 2005-12-08 Rnai Co., Ltd. Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
US7316998B2 (en) 2004-05-27 2008-01-08 Acceleron Pharma Inc. Cerberus/Coco derivatives and uses thereof
EP3228711A1 (en) 2004-06-28 2017-10-11 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US7361468B2 (en) 2004-07-02 2008-04-22 Affymetrix, Inc. Methods for genotyping polymorphisms in humans
EP1618881A1 (en) 2004-07-20 2006-01-25 Santhera Pharmaceuticals (Schweiz) GmbH Use of non-glucocorticoid steroids for the treatment of muscular dystrophy
WO2006017522A2 (en) 2004-08-03 2006-02-16 University Of Utah Research Foundation Use of antisense oligonucleotides to effect translation modulation
US20060099612A1 (en) 2004-09-02 2006-05-11 Suntory Limited Method for analyzing genes of industrial yeasts
CA2582137A1 (en) 2004-10-05 2007-02-15 Wyeth Probe arrays for detecting multiple strains of different species
ITRM20040568A1 (it) 2004-11-18 2005-02-18 Uni Degli Studi Di Roma Tor Vergata Uso della tecnica "phage display" per l'identificazione di peptidi con capacita' di legame a cellule staminali/progenitore, peptidi cosi' ottenuti e loro usi.
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
KR100663277B1 (ko) 2004-12-20 2007-01-02 삼성전자주식회사 휴대단말기의 시스템 관련 이벤트 처리 장치 및 방법
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
CA2596588C (en) 2005-01-31 2017-06-27 University Of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
WO2006108052A2 (en) 2005-04-06 2006-10-12 Genzyme Corporation Peg and polysialic lysosomal enzyme conjugates via acid labile linkers for therapeutic targeting
JP2008538500A (ja) 2005-04-22 2008-10-30 アカデミス ツィーケンホイス ライデン SRタンパク質の結合に対する干渉とRNA二次構造に対する干渉による、mRNA前駆体におけるエクソン認識の調節
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
WO2006121960A2 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
KR100764559B1 (ko) 2005-05-09 2007-10-08 한국표준과학연구원 Epsps 유전자 검색에 활용할 수 있는 fret형광검출용 프로브
ES2397113T3 (es) 2005-06-23 2013-03-04 Isis Pharmaceuticals, Inc. Composiciones y procedimientos para modular el corte y empalme de SMN2
EP2322657B1 (en) 2005-06-28 2014-11-05 Medtronic, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin gene.
DE102005030704A1 (de) 2005-06-29 2007-01-04 Autoliv Development Ab Sicherheitsgurt
ES2435531T3 (es) 2005-07-01 2013-12-20 Index Pharmaceuticals Ab Modulación de la capacidad de respuesta a los esteroides
US7320965B2 (en) 2005-10-28 2008-01-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of Huntingtin gene
US7906617B2 (en) 2005-12-15 2011-03-15 E. I. Du Pont De Nemours And Company Polyethylene binding peptides and methods of use
CA2638908C (en) 2006-01-26 2021-04-27 Susan M. Freier Compositions and their uses directed to huntingtin
SG170819A1 (en) 2006-03-30 2011-05-30 Ptc Therapeutics Inc Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US7855053B2 (en) 2006-07-19 2010-12-21 The Regents Of The University Of California Methods for detecting the presence of expanded CGG repeats in the FMR1 gene 5′ untranslated region
WO2008018795A1 (en) 2006-08-11 2008-02-14 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
AU2007300529A1 (en) 2006-09-27 2008-04-03 Merck Sharp & Dohme Corp. Acylated piperidine derivatives as melanocortin-4 receptor modulators
CA2666185A1 (en) 2006-10-11 2008-04-17 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Influenza targets
CA2666657A1 (en) * 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof
US20100111873A1 (en) 2007-02-20 2010-05-06 Russell Stephen J Treating cancer with viral nucleic acid
PL381824A1 (pl) 2007-02-22 2008-09-01 Instytut Chemii Bioorganicznej Pan W Poznaniu Sekwencja siRNA, wektor, cel molekularny dla reagentów siRNA i wektorów wprowadzanych do komórek i tkanek, sposób oceny specyficzności wyciszania zmutowanego transkryptu, sposób badania oddziaływań enzymów szlaku interferencji RNA z transkryptami oraz zastosowanie sekwencji siRNA i wektora
US20090099066A1 (en) 2007-06-29 2009-04-16 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
CN101790385A (zh) 2007-07-12 2010-07-28 普罗森那技术公司 用于使化合物靶向多种选定器官、组织或肿瘤细胞的分子
WO2009008727A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
US9212205B2 (en) 2007-07-26 2015-12-15 University Of Rochester Nucleic acid binding compounds and methods of use
ES2439591T3 (es) 2007-08-15 2014-01-23 Isis Pharmaceuticals, Inc. Análogos de ácido nucleico de tetrahidropirano
PT2203173E (pt) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
CA2714120A1 (en) 2008-02-08 2009-08-13 Prosensa Holding Bv Methods and means for treating dna repeat instability associated genetic disorders
WO2009101399A1 (en) 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
EP2105145A1 (en) 2008-03-27 2009-09-30 ETH Zürich Method for muscle-specific delivery lipid-conjugated oligonucleotides
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
US8679750B2 (en) 2008-05-09 2014-03-25 The University Of British Columbia Methods and compositions for the treatment of Huntington'S disease
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
WO2009151600A2 (en) 2008-06-10 2009-12-17 Tufts University Smad proteins control drosha-mediated mirna maturation
GB2457965B8 (en) 2008-07-01 2011-02-16 Renovo Ltd Methods and systems for determining efficacy of medicaments.
WO2010006237A2 (en) 2008-07-11 2010-01-14 Alnylam Pharmaceuticals, Inc. Phosphorothioate oligonucleotides non-nucleosidic phosphorothiotes as delivery agents for irna agents
JP2011529686A (ja) 2008-07-29 2011-12-15 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム ポリグルタミンタンパク質発現の選択的阻害
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
CA2777486A1 (en) 2008-10-15 2010-04-22 4S3 Bioscience Inc. Methods and compositions for treatment of myotonic dystrophy
CN105779453A (zh) * 2008-10-24 2016-07-20 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
SI2607484T1 (sl) 2008-10-27 2016-11-30 Biomarin Technologies B.V. Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije
US20100248239A1 (en) 2009-03-24 2010-09-30 Mayo Foundation For Medical Education And Research Methods and materials for detecting fragile x mutations
PL2417257T3 (pl) 2009-04-10 2016-11-30 Antysensowne oligonukleotydy tricyklo-dna, kompozycje i sposoby leczenia choroby
CN102459595A (zh) 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
EA022757B1 (ru) 2009-06-08 2016-02-29 Мираджен Терапьютикс МОТИВЫ ХИМИЧЕСКИХ МОДИФИКАЦИЙ ДЛЯ ИНГИБИТОРОВ И МИМЕТИКОВ мкРНК
JP5809146B2 (ja) 2009-09-11 2015-11-10 アイシス ファーマシューティカルズ, インコーポレーテッド ハンチンチン発現の修飾
JP5963678B2 (ja) * 2009-11-12 2016-08-03 ジ ユニバーシティ オブ ウェスタン オーストラリア 病状を治療するためのアンチセンス分子及び方法
US20110166081A1 (en) 2009-12-03 2011-07-07 University Of Iowa Research Foundation Alpha-dystroglycan as a Protein Therapeutic
WO2011078797A2 (en) 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
JP6141018B2 (ja) 2009-12-24 2017-06-07 バイオマリン テクノロジーズ ベー.フェー. 炎症性障害を治療するための分子
WO2011097641A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
WO2011097614A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Mehods and compositions useful in diseases or conditions related to repeat expansion
CA2799501C (en) 2010-05-28 2022-02-15 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
EP3031920B1 (en) 2010-07-19 2019-08-21 Ionis Pharmaceuticals, Inc. Modulation of dystrophia myotonica-protein kinase (dmpk) expression
CA2806616C (en) 2010-08-20 2015-08-11 Replicor Inc. Oligonucleotide chelate complexes
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
JP2014507143A (ja) 2011-02-08 2014-03-27 ザ シャーロット−メクレンバーグ ホスピタル オーソリティ ドゥーイング/ビジネス/アズ キャロライナズ ヘルスケア システム アンチセンスオリゴヌクレオチド
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
CA3092114A1 (en) 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
WO2012178122A2 (en) * 2011-06-23 2012-12-27 Cold Spring Harbor Laboratory Phenocopy model of disease
WO2013100190A1 (ja) 2011-12-28 2013-07-04 日本新薬株式会社 アンチセンス核酸
CA3120918A1 (en) 2012-01-27 2013-08-01 Biomarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
BR112014026285B1 (pt) 2012-04-23 2021-08-31 Biomarin Technologies B.V Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares
AR091065A1 (es) 2012-05-18 2014-12-30 Replicor Inc Una formulacion farmaceutica que comprende un quelato de oligonucleotidos antiviral para el tratamiento de una infeccion antiviral
BR112014033004B1 (pt) 2012-07-03 2021-10-19 Biomarin Technologies B.V. Oligonucleotídeo para tratamento de pacientes com distrofia muscular

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