JP2016522674A - 遺伝子発現を調節するための組成物及び方法 - Google Patents
遺伝子発現を調節するための組成物及び方法 Download PDFInfo
- Publication number
- JP2016522674A JP2016522674A JP2015512857A JP2015512857A JP2016522674A JP 2016522674 A JP2016522674 A JP 2016522674A JP 2015512857 A JP2015512857 A JP 2015512857A JP 2015512857 A JP2015512857 A JP 2015512857A JP 2016522674 A JP2016522674 A JP 2016522674A
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- nucleotide
- stranded oligonucleotide
- nucleotides
- sequence
- Prior art date
- Legal status (The legal status 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 status listed.)
- Pending
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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
- A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
-
- 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
-
- 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
-
- 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
- C12N2310/315—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
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- 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
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261648077P | 2012-05-16 | 2012-05-16 | |
| US61/648,077 | 2012-05-16 | ||
| PCT/US2013/041437 WO2013173637A1 (en) | 2012-05-16 | 2013-05-16 | Compositions and methods for modulating gene expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016522674A true JP2016522674A (ja) | 2016-08-04 |
| JP2016522674A5 JP2016522674A5 (enExample) | 2016-09-15 |
Family
ID=49584304
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015512857A Pending JP2016522674A (ja) | 2012-05-16 | 2013-05-16 | 遺伝子発現を調節するための組成物及び方法 |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20150141320A1 (enExample) |
| EP (1) | EP2850184A4 (enExample) |
| JP (1) | JP2016522674A (enExample) |
| WO (1) | WO2013173637A1 (enExample) |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2021514182A (ja) * | 2018-02-09 | 2021-06-10 | ジェネンテック, インコーポレイテッド | Tmem106b発現を調節するためのオリゴヌクレオチド |
| JP2023503202A (ja) * | 2020-02-12 | 2023-01-26 | アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー,リミテッド | アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用 |
| JP2023523622A (ja) * | 2020-04-29 | 2023-06-06 | サリオジェン セラピューティクス インコーポレイテッド | 遺伝性黄斑変性症の治療のための組成物及び方法 |
Families Citing this family (87)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN103167883B (zh) | 2010-07-19 | 2016-08-03 | F·C·贝内特 | 肌强直性营养障碍蛋白激酶(dmpk)表达的调节 |
| CA2817256A1 (en) | 2010-11-12 | 2012-05-18 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| AU2013262699A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating ATP2A2 expression |
| US20150152410A1 (en) | 2012-05-16 | 2015-06-04 | Rana Therapeutics, Inc. | Compositions and methods for modulating mecp2 expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| JP2015518710A (ja) | 2012-05-16 | 2015-07-06 | ラナ セラピューティクス インコーポレイテッド | ヘモグロビン遺伝子ファミリー発現を調節するための組成物及び方法 |
| EA201492123A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии семейства генов smn |
| EA201492122A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии utrn |
| ES2709187T3 (es) * | 2013-06-26 | 2019-04-15 | Elanco Us Inc | Marcadores para predecir la resistencia a fármacos de lactona macrocíclica de la Dirofilaria immitis, el agente causal de la enfermedad del gusano del corazón |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| IL300444A (en) | 2013-09-05 | 2023-04-01 | Sarepta Therapeutics Inc | Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase |
| JP2016534035A (ja) | 2013-10-04 | 2016-11-04 | ラナ セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症を治療するための組成物及び方法 |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| WO2015143245A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods for modulating ataxin 2 expression |
| WO2015143246A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| SI3126499T1 (sl) | 2014-04-01 | 2020-09-30 | Biogen Ma Inc. | Sestave za moduliranje izražanja SOD-1 |
| WO2016061487A1 (en) * | 2014-10-17 | 2016-04-21 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof |
| CA2966044A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| WO2016112132A1 (en) * | 2015-01-06 | 2016-07-14 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| BR112017017178A2 (pt) | 2015-02-26 | 2018-04-03 | Ionis Pharmaceuticals Inc | moduladores específicos de alelo de rodopsina de p23h |
| GB201504124D0 (en) | 2015-03-11 | 2015-04-22 | Proqr Therapeutics B V | Oligonucleotides |
| EP3271460A4 (en) | 2015-03-17 | 2019-03-13 | The General Hospital Corporation | RNA INTERACTOM OF POLYCOMB REPRESSIVE COMPLEX 1 (PRC1) |
| WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| US10801024B2 (en) * | 2015-05-20 | 2020-10-13 | Indiana University Research And Technology Corporation | Inhibition of lncRNA HOTAIR and related materials and methods |
| EP3124619B1 (en) * | 2015-07-31 | 2019-03-06 | Menicon Co., Ltd | Reagents, method and kit for across and within dog breed glaucoma diagnosis |
| CA3005247A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of polycystic kidney disease |
| CA3006015A1 (en) | 2015-12-31 | 2017-07-06 | Ionis Pharmaceuticals, Inc. | Methods for reducing ataxin-2 expression |
| CA3012967A1 (en) | 2016-01-29 | 2017-08-03 | Kyowa Hakko Kirin Co., Ltd. | Nucleic acid conjugate |
| WO2017134252A1 (en) * | 2016-02-05 | 2017-08-10 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Antisense oligonucleotides effective to reduce the expression of menin in cancer cells of a subject |
| US11060089B2 (en) | 2016-04-18 | 2021-07-13 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| US10689709B2 (en) | 2016-04-20 | 2020-06-23 | JBS Science Inc. | Kit and method for detecting mutations in CTNNB1 and hTERT, and use thereof in HCC detection and disease management |
| CA3023514A1 (en) | 2016-06-17 | 2017-12-21 | Ionis Pharmaceuticals, Inc. | Modulation of gys1 expression |
| EP3498724B1 (en) | 2016-06-30 | 2023-06-21 | Kyowa Kirin Co., Ltd. | Nucleic acid complex |
| EP3478839A1 (en) | 2016-07-01 | 2019-05-08 | H. Hoffnabb-La Roche Ag | Antisense oligonucleotides for modulating htra1 expression |
| US11655470B2 (en) * | 2016-07-05 | 2023-05-23 | The Usa, As Represented By The Secretary, Dept. Of Health And Human Services | Diagnosing COL6-related disorders and methods for treating same |
| JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
| US11118182B2 (en) | 2016-11-17 | 2021-09-14 | Ramot At Tel-Aviv University Ltd. | Modulators of human KAI1 metastasis suppressor gene, methods and uses thereof |
| EP3548005A4 (en) | 2016-11-29 | 2020-06-17 | Puretech Health LLC | Exosomes for delivery of therapeutic agents |
| WO2018132755A1 (en) | 2017-01-12 | 2018-07-19 | Duke University | Compositions and methods for disrupting the molecular mechanisms associated with mitochondrial dysfunction and neurodegenerative disease |
| EP3604531A4 (en) * | 2017-03-31 | 2020-12-23 | Aichi Medical University | NUCLEIC ACID ANTISENS FOR INHIBITION OF CHONDROITIN SULPHATE BIOSYNTHESIS |
| GB201706009D0 (en) * | 2017-04-13 | 2017-05-31 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of stargardt disease |
| US20190055564A1 (en) * | 2017-06-01 | 2019-02-21 | F. Hoffmann-La Roche Ag | Antisense oligonucleotides for modulating htra1 expression |
| WO2019048631A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | SMALL HNF4A ACTIVATOR RNA COMPOSITIONS AND METHODS OF USE |
| WO2019048645A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE |
| WO2019094315A1 (en) * | 2017-11-08 | 2019-05-16 | Aptamer Diagnostic, Inc. | D-dimer-specific aptamers and methods of use in diagnostics, therapeutic and theranostic purposes |
| TWI809004B (zh) | 2017-11-09 | 2023-07-21 | 美商Ionis製藥公司 | 用於降低snca表現之化合物及方法 |
| PT3717646T (pt) * | 2017-12-01 | 2025-07-28 | Texas A & M Univ Sys | Tratamento antisense da síndrome de angelman |
| MX2020008581A (es) * | 2018-02-21 | 2020-09-21 | Bristol Myers Squibb Co | Oligonucleotidos antisentido de la proteina cinasa del tipo ii delta dependiente del calcio/calmodulina (camk2d) y sus usos. |
| TWI840345B (zh) | 2018-03-02 | 2024-05-01 | 美商Ionis製藥公司 | Irf4表現之調節劑 |
| WO2019169243A1 (en) | 2018-03-02 | 2019-09-06 | Ionis Pharmaceuticals, Inc. | Compounds and methods for the modulation of amyloid-beta precursor protein |
| MX2020010721A (es) | 2018-04-11 | 2020-11-06 | Ionis Pharmaceuticals Inc | Moduladores de la expresion de ezh2. |
| SG11202010215TA (en) | 2018-05-09 | 2020-11-27 | Ionis Pharmaceuticals Inc | Compounds and methods for reducing atxn3 expression |
| TWI851574B (zh) | 2018-05-14 | 2024-08-11 | 美商阿尼拉製藥公司 | 血管收縮素原(AGT)iRNA組成物及其使用方法 |
| WO2020007700A1 (en) * | 2018-07-02 | 2020-01-09 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides targeting spi1 |
| WO2020007772A1 (en) * | 2018-07-02 | 2020-01-09 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides targeting gbp-1 |
| WO2020011653A1 (en) * | 2018-07-09 | 2020-01-16 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides targeting kynu |
| WO2020015959A1 (en) * | 2018-07-19 | 2020-01-23 | Stichting Katholieke Universiteit | Antisense oligonucleotides rescue aberrant splicing of abca4. |
| KR20210038589A (ko) | 2018-07-25 | 2021-04-07 | 아이오니스 파마수티컬즈, 인코포레이티드 | Atxn2 발현 감소용 화합물 및 방법 |
| WO2020084162A1 (en) * | 2018-10-26 | 2020-04-30 | Vrije Universiteit Brussel | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| TW202028222A (zh) | 2018-11-14 | 2020-08-01 | 美商Ionis製藥公司 | Foxp3表現之調節劑 |
| US11214803B2 (en) | 2019-01-31 | 2022-01-04 | Ionis Pharmaceuticals, Inc. | Modulators of YAP1 expression |
| US20200362341A1 (en) * | 2019-03-15 | 2020-11-19 | University Of Massachusetts | Oligonucleotides for tissue specific apoe modulation |
| CN113728104B (zh) * | 2019-03-29 | 2023-10-27 | Ionis制药公司 | 用于调节ube3a-ats的化合物和方法 |
| US11739324B2 (en) | 2019-06-25 | 2023-08-29 | Stichting Katholieke Universiteit | Antisense oligonucleotides rescue aberrant splicing of ABCA4 |
| US20220281911A1 (en) | 2019-08-19 | 2022-09-08 | Mina Therapeutics Limited | Oligonucleotide conjugate compositions and methods of use |
| CA3152513A1 (en) * | 2019-08-30 | 2021-03-04 | Baylor College Of Medicine | System for regulating gene expression |
| EP3822370A1 (en) * | 2019-11-15 | 2021-05-19 | Curiosity Diagnostics Sp. z o.o. | Method of determining the presence of a hyper-virulent clostridioides difficile strain of the b1/nap1/027 group in a sample |
| WO2021126997A1 (en) * | 2019-12-18 | 2021-06-24 | Petdx, Inc. | Methods and compositions for cancer detection, characterization or management in companion animals |
| WO2021153747A1 (ja) * | 2020-01-31 | 2021-08-05 | 株式会社三和化学研究所 | Atn1のアンチセンスオリゴヌクレオチド |
| US20230159925A1 (en) * | 2020-03-04 | 2023-05-25 | Nissan Chemical Corporation | Antisense oligonucleotide of calm2 |
| WO2022006134A2 (en) | 2020-06-29 | 2022-01-06 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating plp1 |
| CA3186629A1 (en) * | 2020-07-22 | 2022-01-27 | The Florey Institute Of Neuroscience And Mental Health | Compositions and methods for treating disorders associated with loss-of-function mutations in scn2a |
| WO2022026589A1 (en) | 2020-07-28 | 2022-02-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing app expression |
| EP4015040A1 (en) * | 2020-12-16 | 2022-06-22 | Fundación para la Formación e Investigación Sanitarias de la Región de Murcia | Method for treating blood diseases |
| KR20230121795A (ko) | 2020-12-18 | 2023-08-21 | 아이오니스 파마수티컬즈, 인코포레이티드 | 인자 xii를 조절하기 위한 화합물 및 방법 |
| WO2022232687A1 (en) * | 2021-04-30 | 2022-11-03 | Greenlight Biosciences, Inc. | Messenger rna therapeutics and compositions |
| IL309217A (en) | 2021-06-18 | 2024-02-01 | Ionis Pharmaceuticals Inc | Compounds and Methods for Reducing IFNAR1 Expression |
| AU2022316149A1 (en) * | 2021-07-21 | 2024-02-08 | AcuraStem Incorporated | Unc13a antisense oligonucleotides |
| US11833221B2 (en) | 2021-09-01 | 2023-12-05 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds for reducing DMPK expression |
| CN118541484A (zh) * | 2021-12-17 | 2024-08-23 | 豪夫迈·罗氏有限公司 | 反义寡核苷酸 |
| WO2023139360A1 (en) * | 2022-01-19 | 2023-07-27 | Autolus Limited | Nucleic acid construct |
| EP4590841A2 (en) * | 2022-09-20 | 2025-07-30 | Aldevron, LLC | Best1 vectors and uses thereof |
| GB202312098D0 (en) * | 2023-08-08 | 2023-09-20 | Ucl Business Ltd | Therapeutic molecules |
| WO2025056973A2 (en) * | 2023-09-13 | 2025-03-20 | Haya Therapeutics Sa | Compositions and methods for modulating wisp2 super-enhancer-associated rna |
| WO2025072672A2 (en) * | 2023-09-27 | 2025-04-03 | Judo Bio, Inc. | Slc6a19-targeting modulatory nucleic acid agents |
| GB202401412D0 (en) * | 2024-02-02 | 2024-03-20 | Harness Therapeutics Ltd | Functional nucleic acid |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPH04320700A (ja) * | 1991-04-17 | 1992-11-11 | Nippon Shoji Kk | アポリポプロテインe遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ |
| WO2008103763A2 (en) * | 2007-02-20 | 2008-08-28 | Sequenom, Inc. | Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation |
| JP2010516256A (ja) * | 2007-01-19 | 2010-05-20 | ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン | Adrb2癌マーカー |
| US20120004278A1 (en) * | 2010-06-18 | 2012-01-05 | The Board Of Trustees Of The Leland Stanford Junior University | Linc rnas in cancer diagnosis and treatment |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008025069A1 (en) * | 2006-08-28 | 2008-03-06 | The Walter And Eliza Hall Institute Of Medical Research | Methods of modulating cellular activity and compositions therefor |
| SG10201506608RA (en) * | 2008-09-26 | 2015-09-29 | Agency Science Tech & Res | 3-deazaneplanocin derivatives |
-
2013
- 2013-05-16 EP EP13790575.8A patent/EP2850184A4/en not_active Withdrawn
- 2013-05-16 US US14/401,248 patent/US20150141320A1/en not_active Abandoned
- 2013-05-16 JP JP2015512857A patent/JP2016522674A/ja active Pending
- 2013-05-16 WO PCT/US2013/041437 patent/WO2013173637A1/en not_active Ceased
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPH04320700A (ja) * | 1991-04-17 | 1992-11-11 | Nippon Shoji Kk | アポリポプロテインe遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ |
| JP2010516256A (ja) * | 2007-01-19 | 2010-05-20 | ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン | Adrb2癌マーカー |
| WO2008103763A2 (en) * | 2007-02-20 | 2008-08-28 | Sequenom, Inc. | Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation |
| US20120004278A1 (en) * | 2010-06-18 | 2012-01-05 | The Board Of Trustees Of The Leland Stanford Junior University | Linc rnas in cancer diagnosis and treatment |
Non-Patent Citations (5)
| Title |
|---|
| KANHERE A. ET AL., MOLECULAR CELL, vol. 38(2010), JPN6017005739, pages 675 - 688, ISSN: 0003506431 * |
| PRENSNER J.R. ET AL., NAT. BIOTECHNOL., vol. 29(8)(2011), JPN6017005745, pages 742 - 749, ISSN: 0003506433 * |
| SARMA K. ET AL., PROC. NATL. ACAD. USA, vol. 107(51)(2010), JPN6017005742, pages 22196 - 22201, ISSN: 0003506430 * |
| ZHAO J. ET AL., SCIENCE, vol. 322(5902)(2008), JPN6017005741, pages 750 - 756, ISSN: 0003506429 * |
| 神武洋二郎 他: "長鎖ncRNAによるクロマチン修飾因子のリクルートメント機構", 実験医学, vol. Vol.29 No.11(2011), JPN7017000587, pages 1716 - 1721, ISSN: 0003506432 * |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2021514182A (ja) * | 2018-02-09 | 2021-06-10 | ジェネンテック, インコーポレイテッド | Tmem106b発現を調節するためのオリゴヌクレオチド |
| JP7281474B2 (ja) | 2018-02-09 | 2023-05-25 | ジェネンテック, インコーポレイテッド | Tmem106b発現を調節するためのオリゴヌクレオチド |
| JP2023503202A (ja) * | 2020-02-12 | 2023-01-26 | アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー,リミテッド | アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用 |
| JP7321614B2 (ja) | 2020-02-12 | 2023-08-07 | アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー,リミテッド | アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用 |
| JP2023523622A (ja) * | 2020-04-29 | 2023-06-06 | サリオジェン セラピューティクス インコーポレイテッド | 遺伝性黄斑変性症の治療のための組成物及び方法 |
Also Published As
| Publication number | Publication date |
|---|---|
| EP2850184A4 (en) | 2016-01-27 |
| US20150141320A1 (en) | 2015-05-21 |
| WO2013173637A1 (en) | 2013-11-21 |
| WO2013173637A8 (en) | 2014-12-24 |
| EP2850184A1 (en) | 2015-03-25 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2016522674A (ja) | 遺伝子発現を調節するための組成物及び方法 | |
| JP2016528873A (ja) | 遺伝子発現を調節するための組成物及び方法 | |
| JP2015518710A (ja) | ヘモグロビン遺伝子ファミリー発現を調節するための組成物及び方法 | |
| JP2015518714A (ja) | 遺伝子発現を調節するための組成物及び方法 | |
| JP2015523853A (ja) | Atp2a2発現を調節するための組成物及び方法 | |
| JP2015518712A (ja) | Mecp2発現を調節するための組成物及び方法 | |
| JP2015518713A (ja) | Utrn発現を調節するための組成物及び方法 | |
| JP2015523855A (ja) | Apoa1及びabca1発現を調節するための組成物及び方法 | |
| ES2586593T3 (es) | Liposomas de retinoide para potenciar la modulación de la expresión de hsp47 | |
| US10041074B2 (en) | Euchromatic region targeting methods for modulating gene expression | |
| JP2015518711A (ja) | Bdnf発現を調節するための組成物及び方法 | |
| JP6129844B2 (ja) | 多量体オリゴヌクレオチド化合物 | |
| JP2015523854A (ja) | Smn遺伝子ファミリー発現を調節するための組成物及び方法 | |
| JP2016521556A (ja) | Foxp3発現を調節するための組成物及び方法 | |
| US20150225722A1 (en) | Methods for selective targeting of heterochromatin forming non-coding rna | |
| JP2016534035A (ja) | 筋萎縮性側索硬化症を治療するための組成物及び方法 | |
| US11464873B2 (en) | RNA-modulating agents | |
| US20180312839A1 (en) | Methods and compositions for increasing smn expression | |
| JP2014527819A5 (enExample) | ||
| KR20180057608A (ko) | 치료용 올리고뉴클레오타이드 | |
| US20180055869A1 (en) | Compositions and methods for modulating rna | |
| US20200054746A1 (en) | Methods for increasing neuronal survival | |
| WO2022026648A1 (en) | Inhibition of incexact1 to treat heart disease | |
| JP7788561B2 (ja) | アンチセンスオリゴヌクレオチド及びその使用 | |
| US20220056443A1 (en) | Metabolic benefits of short mir-22 mirna antagomir therapies |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20150428 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20160513 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20160513 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20170228 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20171012 |