TR201901939T4 - Antisens nükleik asit. - Google Patents

Antisens nükleik asit. Download PDF

Info

Publication number
TR201901939T4
TR201901939T4 TR2019/01939T TR201901939T TR201901939T4 TR 201901939 T4 TR201901939 T4 TR 201901939T4 TR 2019/01939 T TR2019/01939 T TR 2019/01939T TR 201901939 T TR201901939 T TR 201901939T TR 201901939 T4 TR201901939 T4 TR 201901939T4
Authority
TR
Turkey
Prior art keywords
nucleic acid
antisense nucleic
exon
dystrophin gene
human dystrophin
Prior art date
Application number
TR2019/01939T
Other languages
English (en)
Inventor
Wakayama Tatsushi
Seo Haruna
Satou Youhei
Takeda Shin'ichi
Nagata Tetsuya
Original Assignee
Nat Center Neurology & Psychiatry
Nippon Shinyaku Co Ltd
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 Nat Center Neurology & Psychiatry, Nippon Shinyaku Co Ltd filed Critical Nat Center Neurology & Psychiatry
Publication of TR201901939T4 publication Critical patent/TR201901939T4/tr

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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/30—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
    • C12N2310/322—2'-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
    • C12N2310/35—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
    • 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
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

İnsan distrofin geninde ekzon 51'in yüksek verimli bir şekilde atlanmasına olanak sağlayan bir ilaç sağlanmaktadır. Mevcut buluş, insan distrofin geninde ekzon 51'in atlanmasını sağlayan bir antisens oligomeri sağlamaktadır.
TR2019/01939T 2014-03-12 2015-03-11 Antisens nükleik asit. TR201901939T4 (tr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
JP2014048897 2014-03-12

Publications (1)

Publication Number Publication Date
TR201901939T4 true TR201901939T4 (tr) 2019-03-21

Family

ID=54071850

Family Applications (1)

Application Number Title Priority Date Filing Date
TR2019/01939T TR201901939T4 (tr) 2014-03-12 2015-03-11 Antisens nükleik asit.

Country Status (30)

Country Link
US (4) US9988629B2 (tr)
EP (2) EP3118311B1 (tr)
JP (1) JP6606488B2 (tr)
KR (1) KR102335801B1 (tr)
CN (2) CN106459955B (tr)
AU (2) AU2015227733B2 (tr)
BR (1) BR112016020618B1 (tr)
CA (1) CA2939948A1 (tr)
CY (1) CY1121322T1 (tr)
DK (1) DK3118311T3 (tr)
ES (1) ES2710802T3 (tr)
HR (1) HRP20190235T1 (tr)
HU (1) HUE042283T2 (tr)
IL (1) IL247663B (tr)
LT (1) LT3118311T (tr)
MX (1) MX2016011538A (tr)
MY (1) MY193708A (tr)
NZ (1) NZ724836A (tr)
PH (1) PH12016501761B1 (tr)
PL (1) PL3118311T3 (tr)
PT (1) PT3118311T (tr)
RS (1) RS58573B1 (tr)
RU (2) RU2702424C2 (tr)
SG (1) SG11201607095RA (tr)
SI (1) SI3118311T1 (tr)
TR (1) TR201901939T4 (tr)
TW (2) TW201945383A (tr)
UA (1) UA120849C2 (tr)
WO (1) WO2015137409A1 (tr)
ZA (1) ZA201605864B (tr)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2702424C2 (ru) * 2014-03-12 2019-10-08 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
MY185390A (en) 2015-09-15 2021-05-17 Nippon Shinyaku Co Ltd Antisense nucleic acids
WO2017062862A2 (en) 2015-10-09 2017-04-13 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
CA3025575A1 (en) 2016-06-30 2018-01-04 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
WO2018014042A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dystrophin transcript
NZ755416A (en) 2016-12-19 2023-05-26 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
IL267246B2 (en) 2016-12-19 2023-03-01 Sarepta Therapeutics Inc Exon-skipping oligomer conjugates for muscular dystrophy
KR102639633B1 (ko) 2016-12-19 2024-02-26 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
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
WO2019060862A1 (en) * 2017-09-25 2019-03-28 Sarepta Therapeutics, Inc. METHODS FOR PREPARING MORPHOLINO PHOSPHORODIAMIDATE OLIGOMERS VIA FAST FLOW SYNTHESIS
WO2019067975A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3108289A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
MX2021004822A (es) * 2018-11-02 2021-07-06 Biomarin Tech Bv Oligonucleotidos antisentido biespecificos para la omision del exon de la distrofina.
AU2019392928A1 (en) * 2018-12-06 2021-06-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3936135A4 (en) * 2019-01-30 2023-03-15 National Center Of Neurology And Psychiatry NUCLEIC ACID DELIVERY COMPLEX
KR20220145865A (ko) 2020-02-28 2022-10-31 니뽄 신야쿠 가부시키가이샤 엑손 51의 스키핑을 유도하는 안티센스 핵산
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
EP0962463B1 (en) 1989-12-20 2002-07-10 Antivirals Inc. Uncharged morpholino-based polymers having phosphorus-containing chiral intersubunit linkages
JP2924179B2 (ja) 1993-02-19 1999-07-26 日本新薬株式会社 グリセロール誘導体，デバイス及び医薬組成物
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
EP2530156B1 (en) 2002-11-25 2015-11-18 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
DK1766010T3 (da) * 2004-06-28 2011-06-06 Univ Western Australia Antisense-oligonukleotider til induktion af exon-skipping og fremgangsmåder til anvendelse deraf
JPWO2006038608A1 (ja) 2004-10-05 2008-05-15 日本新薬株式会社 オリゴ二本鎖ｒｎａ及び医薬組成物
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
EP1886688A4 (en) 2005-05-30 2013-01-09 Nippon Shinyaku Co Ltd METHOD FOR PRODUCING PREPARATION OF A NUCLEIC ACID-CONTAINING COMPLEX
US8067571B2 (en) * 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
RU2418068C2 (ru) * 2005-08-17 2011-05-10 Сирна Терапьютикс, Инк. Молекулы химически модифицированной короткой интерферирующей нуклеиновой кислоты, которые опосредуют интерференцию рнк
JP2009518006A (ja) * 2005-11-30 2009-05-07 ギルド アソシエイツ， インコーポレイテッド 化学薬品の検出および生分解のための識別的蛍光の酵母バイオセンサー
JP5347510B2 (ja) 2007-02-05 2013-11-20 日本新薬株式会社 ポリエチレングリコール誘導体
HUE028662T2 (en) * 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
CN101861318A (zh) 2007-11-15 2010-10-13 Avi生物制药公司 合成吗啉代低聚物的方法
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
TR201902952T4 (tr) * 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
ES2532634T5 (es) * 2008-10-27 2018-04-30 Biomarin Technologies B.V. Procedimientos y medios para el salto eficiente del exón 45 en el pre-ARNm de la distrofia muscular de Duchenne
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
KR102366851B1 (ko) * 2009-11-12 2022-02-23 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
NZ603606A (en) 2010-05-28 2015-06-26 Sarepta Therapeutics Inc Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
IT1400425B1 (it) * 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
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
CN103619356B (zh) * 2011-05-05 2017-09-12 萨勒普塔医疗公司 肽寡核苷酸缀合物
IN2014DN06220A (tr) * 2011-12-28 2015-10-23 Nippon Shinyaku Co Ltd
RU2702424C2 (ru) * 2014-03-12 2019-10-08 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты

Also Published As

Publication number Publication date
PT3118311T (pt) 2019-02-15
AU2015227733A1 (en) 2016-10-20
HRP20190235T1 (hr) 2019-05-03
RU2730681C2 (ru) 2020-08-24
TWI672314B (zh) 2019-09-21
JPWO2015137409A1 (ja) 2017-04-06
PH12016501761A1 (en) 2016-11-07
RU2019130513A3 (tr) 2020-03-13
CN110951732A (zh) 2020-04-03
AU2020200679B2 (en) 2022-03-10
MY193708A (en) 2022-10-26
LT3118311T (lt) 2019-04-10
EP3118311B1 (en) 2018-12-26
US20220162604A1 (en) 2022-05-26
JP6606488B2 (ja) 2019-11-13
CN106459955B (zh) 2019-12-20
US20180179538A1 (en) 2018-06-28
CY1121322T1 (el) 2020-05-29
US9988629B2 (en) 2018-06-05
ZA201605864B (en) 2018-12-19
EP3514234A1 (en) 2019-07-24
IL247663B (en) 2020-06-30
WO2015137409A1 (ja) 2015-09-17
UA120849C2 (uk) 2020-02-25
CN106459955A (zh) 2017-02-22
ES2710802T3 (es) 2019-04-26
PH12016501761B1 (en) 2016-11-07
TW201540724A (zh) 2015-11-01
RU2702424C2 (ru) 2019-10-08
KR102335801B1 (ko) 2021-12-03
HUE042283T2 (hu) 2019-06-28
CA2939948A1 (en) 2015-09-17
SI3118311T1 (sl) 2019-05-31
BR112016020618B1 (pt) 2023-04-04
RS58573B1 (sr) 2019-05-31
PL3118311T3 (pl) 2019-09-30
AU2020200679A1 (en) 2020-02-20
US20170067048A1 (en) 2017-03-09
MX2016011538A (es) 2017-04-13
EP3118311A4 (en) 2017-10-11
BR112016020618A2 (pt) 2017-08-15
TW201945383A (zh) 2019-12-01
RU2016139108A (ru) 2018-04-13
AU2015227733B2 (en) 2019-10-31
DK3118311T3 (en) 2019-03-11
IL247663A0 (en) 2016-11-30
KR20160132056A (ko) 2016-11-16
EP3118311A1 (en) 2017-01-18
NZ724836A (en) 2022-07-01
RU2019130513A (ru) 2019-10-10
RU2016139108A3 (tr) 2018-08-06
SG11201607095RA (en) 2016-10-28
BR112016020618A8 (pt) 2021-07-13
US11053497B2 (en) 2021-07-06
US20200149040A1 (en) 2020-05-14

Similar Documents

Publication Publication Date Title
TR201901939T4 (tr) Antisens nükleik asit.
IL284423B (en) Antisense oligonucleotides for the treatment of Pompe disease
EP3192869A4 (en) Isolated oligonucleotide and use thereof in nucleic acid sequencing
DK3159409T3 (da) Antisense-nukleinsyre til anvendelse i behandlingen af duchenne muskeldystrofi
IL251390B (en) Polysaccharide and nucleic acid formulations that include viscosity-reducing agents
BR112017008877A2 (pt) métodos e composições para sequenciamento de ácido nucleico-alvo
DK3351633T3 (da) Antisense-nukleinsyre
FR3016768B1 (fr) Utilisation d'une composition enzymatique dans l'alimentation des ruminants
EP3237613A4 (en) Visualizing modified nucleotides and nucleic acid interactions in single cells
MX2022000289A (es) Metodos de evaluacion genomica en ganado.
MA41271A (fr) Méthodes d'utilisation d'oligonucléotides antisens ciblant smad7
MX2018005872A (es) Profarmacos de acido nucleico.
IL254304A0 (en) Nucleoside agents to reduce negative effects of genes involving elongated nucleotide repeats
EP3124622A4 (en) Fluorescent labeled single-stranded nucleic acid and use thereof
PL3145490T3 (pl) Liposomy zawierające kwas dihomo-gamma-linolenowy (dgla), zawierające je preparaty i ich zastosowanie
TH1501003248A (th) ลำดับนิวคลีโอไทด์ที่กำหนดรหัสโปรตีน Homeobox-Leucine Zipper Protein HAT22 (HD-ZIP protein 22) จากพืช Corchorus olitorius เเละพืช Corchorus capsularis เเละวิธีการนำมันมาใช้
TH1501006765A (th) องค์ประกอบน้ำยาปลูกผม
TH1601003100A (th) การใช้อะไมเลสจากแบคทีเรียในอาหารสัตว์สำหรับสัตว์ปีก
TH1601000552A (th) สารยับยั้งของ rorc2 ประเภทเฮเทอโรไบไซโคลแอริล และวิธีการใช้ของมัน
TH182610B (th) กรดนิวคลีอิกแอนทิเซนซ์
TH1401007631A (th) สารองค์ประกอบน้ำยาปลูกผม