HRP20171980T1 - Postupak djelotvornog preskakanja egzona (44) kod duchenneove mišićne distrofije i odgovarajuće sredstvo - Google Patents

Postupak djelotvornog preskakanja egzona (44) kod duchenneove mišićne distrofije i odgovarajuće sredstvo Download PDF

Info

Publication number
HRP20171980T1
HRP20171980T1 HRP20171980TT HRP20171980T HRP20171980T1 HR P20171980 T1 HRP20171980 T1 HR P20171980T1 HR P20171980T T HRP20171980T T HR P20171980TT HR P20171980 T HRP20171980 T HR P20171980T HR P20171980 T1 HRP20171980 T1 HR P20171980T1
Authority
HR
Croatia
Prior art keywords
backbone
oligonucleotide according
oligonucleotide
nucleotide
nucleotide analogue
Prior art date
Application number
HRP20171980TT
Other languages
English (en)
Inventor
Gerardus Johannes Platenburg
Josephus Johannes De Kimpe
Judith Christina Theodora Van Deutekom
Garrit-Jan Boudewijn Van Ommen
Annemieke Aartsma-Rus
Original Assignee
Biomarin Technologies B.V.
Academisch Ziekenhuis Leiden
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Biomarin Technologies B.V., Academisch Ziekenhuis Leiden filed Critical Biomarin Technologies B.V.
Publication of HRP20171980T1 publication Critical patent/HRP20171980T1/hr

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3517—Marker; Tag
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Claims (13)

1. Protusmisleni oligonukleotid kojeg čini slijed 5′ ucagcuucuguuagccacug 3′ (SEQ ID NO:5), naznačen time što je najmanje jedan od nukleotida nukleotidni analog.
2. Oligonukleotid u skladu s patentnim zahtjevom 1, naznačen time što sadrži ostatke RNA, ili jedan ili više ostataka DNA.
3. Oligonukleotid u skladu s patentnim zahtjevom 1, naznačen time što nukleotidni analog ima modificiranu bazu i/ili neprirodnu internukleozidnu vezu, ili kombinaciju tih modifikacija.
4. Oligonukleotid u skladu s patentnim zahtjevom 3, naznačen time što veza između ostataka u okosnici ne uključuje atom fosfora.
5. Oligonukleotid u skladu s bilo kojim od patentnih zahtjeva 1 ili 3 ili 4, naznačen time što navedeni oligonukleotid sadrži najmanje jedan nukleotidni analog s jednim šećernim ostatkom mono- ili disupstituiranim na položaju 2′, 3′ i/ili 5′.
6. Oligonukleotid u skladu s patentnim zahtjevom 3, naznačen time što nukleotidni analog sadrži modificiranu bazu.
7. Oligonukleotid u skladu s patentnim zahtjevom 6, naznačen time što je modificirana baza sintetska ili prirodna baza, poput inozina, ksantina, hipoksantina i drugih -aza, -deaza, -hidroksi, -halogeniranih, -tio, -tiolnih, -alkilnih, -alkenilnih, -alkinil, -tioalkilnih derivata pirimidinskih i purinskih baza.
8. Oligonukleotid u skladu s bilo kojim od patentnih zahtjeva 1-7, naznačen time što sadrži najmanje dva različita tipa nukleotidnih analoga.
9. Oligonukleotid u skladu s patentnim zahtjevom 5, naznačen time što navedeni oligonukleotid sadrži najmanje jedan nukleotidni analog s jedim šećernim ostatkom monosupstituiranim na položaju 2′, 3′ i/ili 5′, po mogućnosti na 2′-položaju, a poželjnije gdje je šećerni ostatak 2′-metoksi supstituiran.
10. Oligonukleotid u skladu s patentnim zahtjevom 5, naznačen time što je nukleotidni analog LNA (blokirana nukleinska kiselina).
11. Oligonukleotid u skladu s patentnim zahtjevom 4, naznačen time što se modificiranu okosnicu bira iz skupine koju čine morfolino okosnica, karbamatna okosnica, siloksanska okosnica, sulfidna okosnica, sulfoksidna okosnica, sulfonska okosnica, formacetilna okosnica, tioformacetilna okosnica, metilenformacetilna okosnica, riboacetilna okosnica, okosnica koja sadrži alken, sulfamatna okosnica, sulfonatna okosnica, sulfonamidna okosnica, metilenimino okosnica, metilenhidrazino okosnica i amidna okosnica.
12. Farmaceutski pripravak, naznačen time što sadrži oligonukleotid u skladu s bilo kojim od patentnih zahtjeva 1-11, kao i farmaceutski prihvatljiv nosač.
13. Oligonukleotid u skladu s bilo kojim od patentnih zahtjeva 1-11, ili farmaceutski pripravak u skladu s patentnim zahtjevom 12, naznačeni time što su namijenjeni upotrebi kao medikament za liječenje pacijenta s DMD-om ili BMD-om.
HRP20171980TT 2008-05-14 2017-12-20 Postupak djelotvornog preskakanja egzona (44) kod duchenneove mišićne distrofije i odgovarajuće sredstvo HRP20171980T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP08156193A EP2119783A1 (en) 2008-05-14 2008-05-14 Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US12801008P 2008-05-15 2008-05-15
EP14179979.1A EP2813570B1 (en) 2008-05-14 2009-05-14 Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means

Publications (1)

Publication Number Publication Date
HRP20171980T1 true HRP20171980T1 (hr) 2018-02-09

Family

ID=39800733

Family Applications (2)

Application Number Title Priority Date Filing Date
HRP20151053TT HRP20151053T1 (hr) 2008-05-14 2015-10-05 Metoda za uäśinkovito preskakanje eksona (44) u duchenne mišiä†noj distrofiji i srodnim namjenama
HRP20171980TT HRP20171980T1 (hr) 2008-05-14 2017-12-20 Postupak djelotvornog preskakanja egzona (44) kod duchenneove mišićne distrofije i odgovarajuće sredstvo

Family Applications Before (1)

Application Number Title Priority Date Filing Date
HRP20151053TT HRP20151053T1 (hr) 2008-05-14 2015-10-05 Metoda za uäśinkovito preskakanje eksona (44) u duchenne mišiä†noj distrofiji i srodnim namjenama

Country Status (18)

Country Link
US (4) US9139828B2 (hr)
EP (5) EP2119783A1 (hr)
JP (2) JP5696039B2 (hr)
CN (1) CN102171342B (hr)
AU (5) AU2009247037B2 (hr)
CA (2) CA2723968C (hr)
CY (1) CY1117098T1 (hr)
DK (2) DK2813570T3 (hr)
ES (3) ES2656904T3 (hr)
HK (3) HK1154620A1 (hr)
HR (2) HRP20151053T1 (hr)
HU (2) HUE038111T2 (hr)
IL (5) IL300399A (hr)
LT (1) LT2813570T (hr)
NZ (2) NZ600183A (hr)
PL (2) PL2283134T3 (hr)
PT (2) PT2813570T (hr)
WO (1) WO2009139630A2 (hr)

Families Citing this family (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US7807816B2 (en) 2004-06-28 2010-10-05 University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
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
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2350281B1 (en) 2008-10-24 2014-05-14 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
EP3305302B1 (en) 2009-06-17 2018-09-19 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
HUE040445T2 (hu) 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
EP2516647B1 (en) 2009-12-24 2016-12-14 BioMarin Technologies B.V. Molecule for treating an inflammatory disorder
EP2582397A4 (en) * 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR MODULATING THE INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US20140088174A1 (en) 2011-04-05 2014-03-27 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Compounds and methods for altering activin receptor-like kinase signaling
WO2012138487A2 (en) * 2011-04-07 2012-10-11 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
JP6141770B2 (ja) 2011-12-28 2017-06-07 日本新薬株式会社 アンチセンス核酸
BR112014018427B1 (pt) 2012-01-27 2021-11-03 Biomarin Technologies B.V. Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker
JP6460983B2 (ja) * 2012-07-03 2019-01-30 バイオマリン テクノロジーズ ベー．フェー． 筋ジストロフィー患者の治療のためのオリゴヌクレオチド
EP2943225A4 (en) * 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT
BR122020016865B1 (pt) 2013-03-14 2022-12-27 Sarepta Therapeutics, Inc. Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
CA2906812A1 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
EP3797780B1 (en) 2014-04-17 2022-09-14 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CA2951221A1 (en) 2014-06-17 2015-12-23 Nippon Shinyaku Co., Ltd. Antisense nucleic acids
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
EP3085785A1 (en) * 2015-04-22 2016-10-26 Université de Strasbourg Dynamin 2 inhibitor for the treatment of duchenne's muscular dystrophy
EP3307891B8 (en) * 2015-06-10 2019-11-13 Association Institut de Myologie Combined therapy for duchenne muscular dystrophy
UA123359C2 (uk) 2015-09-15 2021-03-24 Ніппон Шин'Яку Ко., Лтд. Антисенсова нуклеїнова кислота
TW201722439A (zh) 2015-10-09 2017-07-01 波濤生命科學有限公司 寡核苷酸組合物及其方法
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
MA45618A (fr) 2016-06-30 2019-05-08 Sarepta Therapeutics Inc Sauts d'exons oligomères pour la dystrophie musculaire
BR112019000195A2 (pt) 2016-07-07 2019-04-24 Rubius Therapeutics, Inc. composições e métodos relacionados a sistemas celulares terapêuticos que expressam rna exógeno
JP7416406B2 (ja) * 2016-12-08 2024-01-17 ケース ウェスタン リザーブ ユニバーシティ 機能的ミエリン産生を増進させるための方法および組成物
SG10202100491QA (en) 2016-12-19 2021-02-25 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
CN110636866A (zh) 2016-12-19 2019-12-31 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
HUE059843T2 (hu) 2016-12-19 2023-01-28 Sarepta Therapeutics Inc Exonátugró oligomerkonjugátumok izomdisztrófiára
JP2020505330A (ja) 2017-01-06 2020-02-20 アビディティー バイオサイエンシーズ エルエルシー エクソンスキッピングを誘導する核酸ポリペプチド組成物および方法
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
AU2018335880A1 (en) * 2017-09-22 2020-04-16 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and methods of inducing exon skipping
WO2019113393A1 (en) 2017-12-06 2019-06-13 Avidity Biosciences Llc Compositions and methods of treating muscle atrophy and myotonic dystrophy
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
CN110760511B (zh) * 2018-07-27 2021-09-07 广东赤萌医疗科技有限公司 一种用于治疗杜氏肌营养不良症的gRNA、表达载体、CRISPR-Cas9***
US20210308273A1 (en) * 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN114466682A (zh) * 2019-08-02 2022-05-10 全国儿童医院研究所 用于治疗基于肌营养不良蛋白的肌病的靶向外显子44的核酸和包含所述核酸的重组腺相关病毒
IL293997A (en) 2019-12-19 2022-08-01 Nippon Shinyaku Co Ltd Antistrand nucleic acids that allow exon skipping
KR20220148230A (ko) 2020-02-28 2022-11-04 아이오니스 파마수티컬즈, 인코포레이티드 Smn2를 조절하기 위한 화합물 및 방법
AU2021237465A1 (en) 2020-03-19 2022-10-13 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
WO2021242674A1 (en) * 2020-05-26 2021-12-02 Ptc Therapeutics, Inc. Quantification of dystrophin
CN112063621B (zh) * 2020-09-02 2022-06-28 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
EP4299744A1 (en) * 2021-02-25 2024-01-03 Osaka University Oligonucleotide for inducing n-exon skipping during rest mrna precursor processing
JP2024518068A (ja) 2021-05-10 2024-04-24 エントラーダ セラピューティクス，インコーポレイティド 細胞内治療のための組成物及び方法
WO2022270585A1 (ja) 2021-06-23 2022-12-29 日本新薬株式会社 アンチセンスオリゴマーの組み合わせ
KR20240038967A (ko) 2021-06-23 2024-03-26 엔트라다 테라퓨틱스, 인크. Cug 반복을 표적화하기 위한 안티센스 화합물 및 방법
US11912779B2 (en) 2021-09-16 2024-02-27 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases

Family Cites Families (200)

* 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.
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
EP0558697A1 (en) 1991-06-28 1993-09-08 Massachusetts Institute Of Technology Localized oligonucleotide therapy
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
EP1897942A1 (en) 1993-05-11 2008-03-12 The University Of North Carolina At Chapel Hill Antisense oligonucleotides which combat aberrant splicing and methods of using the same
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
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
SK109198A3 (en) 1996-02-14 1999-06-11 Isis Pharmaceuticals Inc Sugar-modified gapped oligonucleotides
US6251589B1 (en) 1996-07-18 2001-06-26 Srl, Inc. Method for diagnosing spinocerebellar ataxia type 2 and primers therefor
WO1998018920A1 (fr) 1996-10-30 1998-05-07 Srl, Inc. Fragments d'adnc du gene responsable de l'ataxie spino-cerebelleuse de 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
AU7265298A (en) 1997-04-29 1998-11-24 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
WO1999016871A2 (en) 1997-09-22 1999-04-08 Max-Planck-Gesellschaft Zur Forderung Der Wissensc 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
US6322978B1 (en) 1998-04-20 2001-11-27 Joslin Diabetes Center, Inc. Repeat polymorphism in the frataxin gene and uses therefore
AU751480B2 (en) 1998-04-29 2002-08-15 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into ribozymes
WO1999063975A2 (en) 1998-06-10 1999-12-16 Biognostik Gesellschaft für Biomolekulare Diagnostik mbH A method for stimulating the immune system
US6924355B2 (en) 1998-09-01 2005-08-02 Genentech, Inc. PRO1343 polypeptides
CA2343934A1 (en) 1998-09-25 2000-04-06 The Children's Medical Center Corporation Short peptides which selectively modulate the activity of protein kinases
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
AU771579B2 (en) 1998-10-26 2004-03-25 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
AU777499B2 (en) 1999-04-08 2004-10-21 Gen-Probe Incorporated 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
CA2386540A1 (en) 1999-10-04 2001-04-12 University Of Medicine And Dentistry Of New Jersey Novel carbamates and ureas
US6165786A (en) 1999-11-03 2000-12-26 Isis Pharmaceuticals, Inc. Antisense modulation of nucleolin expression
AU3497701A (en) 2000-02-08 2001-08-20 Ribozyme Pharmaceuticals, Inc. Nucleozymes with endonuclease activity
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
WO2001079283A1 (en) 2000-04-13 2001-10-25 University Of British Columbia 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
CA2407309C (en) 2000-04-28 2011-08-02 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
CA2407942A1 (en) 2000-05-01 2001-11-08 Hybridon, Inc. Modulation of oligonucleotide cpg-mediated immune stimulation by positional modification of nucleosides
US20020013287A1 (en) 2000-05-09 2002-01-31 Reliable Biopharmaceuticals, Inc. St Louis Missouri 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
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
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
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
AU2001296600A1 (en) 2000-10-06 2002-04-15 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
CA2449412C (en) 2000-11-30 2013-04-02 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
US20070021360A1 (en) 2001-04-24 2007-01-25 Nyce Jonathan W Compositions, formulations and kit with anti-sense oligonucleotide and anti-inflammatory steroid and/or obiquinone for treatment of respiratory and lung disesase
CA2414782C (en) 2001-05-11 2012-10-09 Regents Of The University Of Minnesota Intron associated with myotonic dystrophy type 2 and methods of use
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)
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)
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
CA2451128A1 (en) 2001-06-26 2003-01-09 Bristol-Myers Squibb Company N-heterocyclic inhibitors of tnf-alpha expression
DK1406667T3 (da) 2001-07-06 2008-06-16 Topigen Pharmaceuticals Inc Fremgangsmåder til at öge oligonukleotiders in vivo-effektivitet og hæmme inflammation hos pattedyr
AU2002326589B2 (en) 2001-08-07 2008-06-05 University Of Delaware Compositions and methods for the prevention and treatment of Huntington's disease
WO2003014145A2 (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
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
WO2003062258A1 (en) 2002-01-22 2003-07-31 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)
WO2003074551A2 (en) 2002-03-01 2003-09-12 The Administrators Of The Tulane Educational Fund Conjugates of therapeutic or cytotoxic agents and biologically active peptides
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
CA2493297A1 (en) 2002-08-12 2004-02-19 Universite De Sherbrooke Methods to reprogram splice site selection in pre-messenger rnas
GB0219143D0 (en) 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
WO2004037854A1 (en) 2002-10-23 2004-05-06 Centre For Research And Technology Hellas/Institute Of Agrobiotechnology In.A Prion protein-binding peptide sequences
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
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
EP2314691A3 (en) * 2002-11-14 2012-01-18 Dharmacon, Inc. Fuctional and hyperfunctional siRNA
CA2796924C (en) 2002-11-25 2016-12-13 Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
GB0228079D0 (en) 2002-12-02 2003-01-08 Laxdale Ltd Huntington's Disease
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
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
US20040198640A1 (en) * 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
US7514551B2 (en) 2003-04-03 2009-04-07 Enzo Life Sciences, Inc. Multisignal labeling reagents, and processes and uses therefor
DK2351844T3 (da) * 2003-04-29 2014-09-22 Sarepta Therapeutics Inc Præparater til forøgelse af transport- og antisense-effektivitet af nukleinsyreanalog i celler
US7589189B2 (en) 2003-05-14 2009-09-15 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
EP1635870A2 (en) 2003-06-02 2006-03-22 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
WO2005086768A2 (en) 2004-03-11 2005-09-22 Albert Einstein College Of Medicine Of Yeshiva University 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)
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
CA2567810A1 (en) 2004-05-27 2005-12-08 Acceleron Pharma Inc. Cerberus/coco derivatives and uses thereof
US7807816B2 (en) 2004-06-28 2010-10-05 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
EP1817432A2 (en) 2004-10-05 2007-08-15 Wyeth a Corporation of the State of Delaware 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
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
EP1844148A2 (en) 2005-01-31 2007-10-17 University of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
US7341720B2 (en) 2005-04-06 2008-03-11 Genzyme Corporation Targeting of glycoprotein therapeutics
US20090312532A1 (en) 2005-04-22 2009-12-17 Van Deutekom Judith Christina Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secodary rna structure
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
ATE522626T1 (de) 2005-06-28 2011-09-15 Medtronic Inc Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken
ES2435531T3 (es) 2005-07-01 2013-12-20 Index Pharmaceuticals Ab Modulación de la capacidad de respuesta a los esteroides
WO2007058894A2 (en) * 2005-11-10 2007-05-24 The University Of North Carolina At Chapel Hill Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
US7906617B2 (en) 2005-12-15 2011-03-15 E. I. Du Pont De Nemours And Company Polyethylene binding peptides and methods of use
EP2428227B1 (en) 2006-01-26 2016-06-22 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
US7901882B2 (en) * 2006-03-31 2011-03-08 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
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
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
WO2008039418A2 (en) 2006-09-27 2008-04-03 Merck & Co., Inc. Acylated piperidine derivatives as melanocortin-4 receptor modulators
SG166778A1 (en) 2006-10-11 2010-12-29 Max Planck Gesellschaft Influenza targets
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
ES2694726T3 (es) * 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Conjugados peptídicos específicos de tejido y métodos
AU2008273094B2 (en) 2007-07-12 2013-05-09 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
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
AU2008317566B2 (en) * 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
NZ587178A (en) 2008-02-08 2011-11-25 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
EP2297341A4 (en) 2008-05-09 2013-01-09 Univ British Columbia METHOD AND COMPOSITIONS FOR TREATING MORBUS HUNTINGTON
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
WO2009144481A2 (en) 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
WO2009151600A2 (en) 2008-06-10 2009-12-17 Tufts University Smad proteins control drosha-mediated mirna maturation
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2346905A4 (en) 2008-10-15 2012-03-07 4S3 Bioscience Inc METHODS AND COMPOSITIONS FOR TREATING MYOTONIC DYSTROPHY
EP2350281B1 (en) 2008-10-24 2014-05-14 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
CA2741629C (en) 2008-10-27 2022-07-05 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna
CN105296474B (zh) 2009-03-24 2020-05-12 奥斯瑞根公司 对fmr1和fmr2基因5’非翻译区进行表征的pcr方法
US20100248239A1 (en) 2009-03-24 2010-09-30 Mayo Foundation For Medical Education And Research Methods and materials for detecting fragile x mutations
WO2010115993A1 (en) 2009-04-10 2010-10-14 Association Institut De Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
US20120046342A1 (en) 2009-04-24 2012-02-23 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
HUE033113T2 (en) 2009-09-11 2017-11-28 Ionis Pharmaceuticals Inc Modifying Huntingtin expression
HUE040445T2 (hu) 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
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
EP2516647B1 (en) 2009-12-24 2016-12-14 BioMarin Technologies B.V. Molecule for treating an inflammatory disorder
US20130059902A1 (en) 2010-02-08 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
EP2605794B1 (en) 2010-08-20 2016-07-20 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
WO2012109296A1 (en) 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
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
KR102183273B1 (ko) 2011-05-05 2020-11-27 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
JP6141770B2 (ja) 2011-12-28 2017-06-07 日本新薬株式会社 アンチセンス核酸
BR112014018427B1 (pt) 2012-01-27 2021-11-03 Biomarin Technologies B.V. Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker
CN104271741A (zh) 2012-04-23 2015-01-07 普罗森萨科技有限公司 用于治疗神经肌肉障碍的具有改善特性的rna调节寡核苷酸
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
JP6460983B2 (ja) 2012-07-03 2019-01-30 バイオマリン テクノロジーズ ベー．フェー． 筋ジストロフィー患者の治療のためのオリゴヌクレオチド

Also Published As

Publication number Publication date
US20190292541A1 (en) 2019-09-26
IL300399A (en) 2023-04-01
DK2283134T3 (en) 2015-11-23
CN102171342A (zh) 2011-08-31
CA3048624A1 (en) 2009-11-19
EP2119783A1 (en) 2009-11-18
HRP20151053T1 (hr) 2016-01-29
IL209301A0 (en) 2011-01-31
EP2283134B1 (en) 2015-09-30
DK2813570T3 (en) 2017-12-11
US20160053262A1 (en) 2016-02-25
PL2283134T3 (pl) 2016-03-31
EP2813570A1 (en) 2014-12-17
JP5891268B2 (ja) 2016-03-22
US20210207138A1 (en) 2021-07-08
IL282772B1 (en) 2023-03-01
PT2283134E (pt) 2015-12-24
CN102171342B (zh) 2014-07-16
EP3872178A1 (en) 2021-09-01
CA2723968C (en) 2019-07-30
IL253750A0 (en) 2017-09-28
AU2019283969A1 (en) 2020-01-23
IL209301A (en) 2017-08-31
NZ589230A (en) 2012-09-28
PT2813570T (pt) 2018-02-12
WO2009139630A9 (en) 2010-05-06
AU2017268534B2 (en) 2019-10-03
EP2813570B1 (en) 2017-11-15
US10246707B2 (en) 2019-04-02
IL282772B2 (en) 2023-07-01
AU2017268534A1 (en) 2017-12-21
HK1251258A1 (zh) 2019-01-25
JP2011520444A (ja) 2011-07-21
JP2014221058A (ja) 2014-11-27
EP3284823A2 (en) 2018-02-21
EP3284823A3 (en) 2018-03-14
LT2813570T (lt) 2018-04-10
AU2009247037A1 (en) 2009-11-19
CA2723968A1 (en) 2009-11-19
HUE026385T2 (en) 2016-06-28
WO2009139630A2 (en) 2009-11-19
HK1154620A1 (zh) 2012-04-27
EP3284823B1 (en) 2021-03-31
IL268242A (en) 2019-09-26
HUE038111T2 (hu) 2018-09-28
AU2019283969B2 (en) 2022-03-17
NZ600183A (en) 2013-03-28
CY1117098T1 (el) 2017-04-05
HK1204004A1 (en) 2015-11-06
ES2551154T3 (es) 2015-11-16
ES2867809T3 (es) 2021-10-20
JP5696039B2 (ja) 2015-04-08
IL282772A (en) 2021-06-30
US9139828B2 (en) 2015-09-22
AU2015252074A1 (en) 2015-11-19
WO2009139630A3 (en) 2010-03-25
AU2022201136A1 (en) 2022-03-17
AU2009247037B2 (en) 2016-02-04
PL2813570T3 (pl) 2018-04-30
ES2656904T3 (es) 2018-02-28
US20120059042A1 (en) 2012-03-08
EP2283134A2 (en) 2011-02-16
IL268242B (en) 2021-05-31

Similar Documents

Publication Publication Date Title
HRP20171980T1 (hr) Postupak djelotvornog preskakanja egzona (44) kod duchenneove mišićne distrofije i odgovarajuće sredstvo
Porfiri et al. Juvenile specimen of Megaraptor (Dinosauria, Theropoda) sheds light about tyrannosauroid radiation
HRP20160078T1 (hr) METODE I SREDSTVA ZA UČINKOVITO PRESKAKANJE EKSONA 45 U pre-mRNA DUCHENNE MIŠIĆNE DISTROFIJE
JP2019518037A (ja) Ｂ型肝炎感染症治療用のＰＡＰＤ５又はＰＡＰＤ７のｍＲＮＡ減少用核酸分子
ES2595079T3 (es) Ácido ribonucleico de interferencia corto (ARNip)
JP2011520444A5 (hr)
RU2018119315A (ru) Олигонуклеотиды для индуцирования отцовской экспрессии ube3a
HRP20110352T1 (hr) Antisens olinukleotidi za induciranje zaobilaženja egzona i postupci njihove upotrebe
JP2006512929A5 (hr)
RU2014117018A (ru) Аптамер против ngf и его применение
JP2017536119A5 (hr)
JP2016502858A5 (hr)
CA2520541A1 (en) Antisense oligonucleotides (odn) against smad7 and uses thereof in medical field
JP2016116520A5 (hr)
RU2016146819A (ru) Композиции и способы модулирования экспрессии рецептора гормона роста
JP2016523087A5 (hr)
US10758558B2 (en) Hybrid oligonucleotides and uses thereof
JP2010514768A5 (hr)
JP2013518603A5 (hr)
EP2271351A2 (en) Compositions and methods for treating viral infections
CN103298475A (zh) 作为治疗剂的寡核苷酸类似物的设计
HRP20221378T1 (hr) Spojevi i postupci za modulaciju smn2
US20180030452A1 (en) Targeting oligonucleotides and uses thereof to modulate gene expression
WO2008082440A3 (en) Fluorescent nucleoside analogues
Norell et al. The earliest iguanine lizard (Reptilia: Squamata) and its bearing on iguanine phylogeny