MX340397B - Modulacion de expresion de huntingtina. - Google Patents
Modulacion de expresion de huntingtina.Info
- Publication number
- MX340397B MX340397B MX2012003006A MX2012003006A MX340397B MX 340397 B MX340397 B MX 340397B MX 2012003006 A MX2012003006 A MX 2012003006A MX 2012003006 A MX2012003006 A MX 2012003006A MX 340397 B MX340397 B MX 340397B
- Authority
- MX
- Mexico
- Prior art keywords
- modulation
- huntingtin expression
- compositions
- compounds
- methods
- Prior art date
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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P15/00—Drugs for genital or sexual disorders; Contraceptives
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
- A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
-
- 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
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/20—Hypnotics; Sedatives
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/22—Anxiolytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/24—Antidepressants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- 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
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone 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
- C12N2310/34—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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Genetics & Genomics (AREA)
- Neurology (AREA)
- Molecular Biology (AREA)
- Neurosurgery (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Diabetes (AREA)
- Physical Education & Sports Medicine (AREA)
- Psychiatry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Cardiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Hospice & Palliative Care (AREA)
- Endocrinology (AREA)
- Pain & Pain Management (AREA)
- Rheumatology (AREA)
- Heart & Thoracic Surgery (AREA)
- Hematology (AREA)
Abstract
La presente invención se refiere a métodos, compuestos y composiciones para reducir la expresión de ARNm y proteína de huntingtina en un animal. Dichos métodos, compuestos, y composiciones son útiles para tratar, prevenir, retrasar, o mejorar la enfermedad de Huntington, o un síntoma de la misma. Un oligonucleótido de cadena individual dirigido a un ácido nucleico que codifica para huntingtina y que puede inhibir la expresión de huntingtina, en el cual el oligonucleótido modificado comprende: un segmento de espacio que consiste de diez desoxinucleósidos ligados; un segmento de ala 5´que consiste de cinco nucleósidos ligados; y un segmento de la 3´que consiste de cinco nucleósidos ligados; en el cual el segmento de espacio está posicionado entre el segmento de ala 5´y el segmento de la 3´, en el cual cada nucleósido de cada segmento de ala comprende un azúcar 2´-O-metoxietilado; en el cual los enlazamientos internucleósido dentro del segmento de espacio, los enlazamientos que conectan el segmento de espacio al segmento de ala 5´o 3´, y los enlazamientos para los nucleósidos más hacia el extremo 5´y más hacia el extremo 3´de cada segmento de ala son todos enlazamientos de fosforotioato, los enlazamientos internucleósido que conectan el resto de los nucleósidos de los segmentos de ala tanto 5´como 3´son enlazamientos de fosfoldiéster; y en el cual todas la scitosinas son 5-metilcitosinas; y en donde la secuencia de nucleobase del iligonucleótido consiste de una secuencia recitada en SEQ ID NO: 22 ó 32.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US24185309P | 2009-09-11 | 2009-09-11 | |
PCT/US2010/048532 WO2011032045A1 (en) | 2009-09-11 | 2010-09-10 | Modulation of huntingtin expression |
Publications (2)
Publication Number | Publication Date |
---|---|
MX2012003006A MX2012003006A (es) | 2012-06-28 |
MX340397B true MX340397B (es) | 2016-07-07 |
Family
ID=43732828
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2012003006A MX340397B (es) | 2009-09-11 | 2010-09-10 | Modulacion de expresion de huntingtina. |
MX2016004031A MX353152B (es) | 2009-09-11 | 2012-03-09 | Modulación de expresión de huntingtina. |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2016004031A MX353152B (es) | 2009-09-11 | 2012-03-09 | Modulación de expresión de huntingtina. |
Country Status (19)
Country | Link |
---|---|
US (8) | US8906873B2 (es) |
EP (3) | EP3178933B1 (es) |
JP (5) | JP5809146B2 (es) |
KR (5) | KR101774526B1 (es) |
CN (3) | CN111705058A (es) |
AU (4) | AU2010292003B2 (es) |
BR (1) | BR112012005448B1 (es) |
CA (2) | CA2773886C (es) |
DK (1) | DK2475675T3 (es) |
ES (2) | ES2615534T3 (es) |
HU (1) | HUE033113T2 (es) |
IL (4) | IL218482A (es) |
MX (2) | MX340397B (es) |
NZ (3) | NZ714887A (es) |
PL (1) | PL2475675T3 (es) |
PT (1) | PT2475675T (es) |
RU (2) | RU2562861C2 (es) |
SI (1) | SI2475675T1 (es) |
WO (1) | WO2011032045A1 (es) |
Families Citing this family (44)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2614827B1 (en) | 2007-10-26 | 2017-06-28 | 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 |
EP2317847B1 (en) | 2008-07-29 | 2019-04-17 | The Board of Regents of The University of Texas System | Selective inhibition of polyglutamine protein expression |
BRPI0923225A2 (pt) | 2008-12-02 | 2016-10-04 | Chiralgen Ltd | metodo para sintese de acidos nucleicos modificados no atomo de fosforo |
KR101885383B1 (ko) | 2009-07-06 | 2018-08-03 | 웨이브 라이프 사이언시스 리미티드 | 신규한 핵산 프로드러그 및 그의 사용 방법 |
JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
US8569254B2 (en) | 2010-12-10 | 2013-10-29 | National Yang Ming University | Methods for modulating the expression and aggregation of CAG-expanded gene product in cells and methods for identifying agents useful for doing the same |
AU2012246822B2 (en) * | 2011-04-22 | 2017-05-25 | Biomarin Technologies B.V. | New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (DM1) |
CA2839437A1 (en) * | 2011-06-16 | 2012-12-20 | Isis Pharmaceuticals, Inc. | Antisense modulation of fibroblast growth factor receptor 4 expression |
CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
EP4043039A1 (en) | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
WO2013159108A2 (en) * | 2012-04-20 | 2013-10-24 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
EP4219516A3 (en) | 2012-07-13 | 2024-01-10 | Wave Life Sciences Ltd. | Chiral control |
EP2873674B1 (en) | 2012-07-13 | 2020-05-06 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
EP2906255B1 (en) | 2012-10-12 | 2023-02-22 | Ionis Pharmaceuticals, Inc. | Antisense compounds and uses thereof |
TW202246503A (zh) * | 2013-07-19 | 2022-12-01 | 美商百健Ma公司 | 用於調節τ蛋白表現之組合物 |
CN103601798B (zh) * | 2013-11-11 | 2015-11-18 | 杭州璞题生物科技有限公司 | 亨廷顿蛋白的酰基化修饰方法 |
JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
KR102423317B1 (ko) | 2014-01-16 | 2022-07-22 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
CA2978521A1 (en) | 2015-05-29 | 2016-12-08 | National Yang-Ming University | Nucleoside agents for the reduction of the deleterious activity of extended nucleotide repeat containing genes |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
MX2021001091A (es) | 2015-12-10 | 2022-04-26 | Ptc Therapeutics Inc | Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington. |
EP3881857A1 (en) | 2016-02-18 | 2021-09-22 | The Penn State Research Foundation | Generating gabaergic neurons in brains |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
WO2018102397A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
US11407753B2 (en) | 2017-06-05 | 2022-08-09 | Ptc Therapeutics, Inc. | Compounds for treating Huntington's disease |
BR112019027719A2 (pt) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics, Inc. | métodos para tratar a doença de huntington |
US11382918B2 (en) | 2017-06-28 | 2022-07-12 | Ptc Therapeutics, Inc. | Methods for treating Huntington's Disease |
JP7399870B2 (ja) | 2018-03-27 | 2023-12-18 | ピーティーシー セラピューティクス, インコーポレイテッド | ハンチントン病を処置するための化合物 |
JP7421507B2 (ja) | 2018-06-27 | 2024-01-24 | ピーティーシー セラピューティクス, インコーポレイテッド | ハンチントン病を処置するためのヘテロ環式およびヘテロアリール化合物 |
PL3864163T3 (pl) | 2018-10-09 | 2024-05-20 | The University Of British Columbia | Kompozycje i układy zawierające pęcherzyki zdolne do transfekcji wolne od rozpuszczalników organicznych i detergentów oraz związane z nimi sposoby postępowania |
CA3145397A1 (en) * | 2019-06-28 | 2020-12-30 | The Penn State Research Foundation | Methods and materials for treating huntington's disease |
CA3156848A1 (en) | 2019-11-01 | 2021-05-06 | Novartis Ag | The use of a splicing modulator for a treatment slowing progression of huntington's disease |
TW202132567A (zh) * | 2019-11-01 | 2021-09-01 | 美商阿尼拉製藥公司 | 亨汀頓蛋白(HTT)iRNA劑組成物及其使用方法 |
CA3172336A1 (en) * | 2020-02-21 | 2021-08-26 | Ionis Pharmaceuticals, Inc | Methods for reducing htt expression |
US20220090069A1 (en) * | 2020-08-03 | 2022-03-24 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
WO2022165122A1 (en) * | 2021-01-29 | 2022-08-04 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating huntingtin |
TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
CN114150055A (zh) * | 2021-12-06 | 2022-03-08 | 昆明医科大学 | 一种以减少滑移峰的产生提高亨廷顿舞蹈症基因检测准确率的试剂盒及其制备方法 |
WO2024064954A2 (en) * | 2022-09-23 | 2024-03-28 | The Children's Hospital Of Philadelphia | Compositions and methods for treating huntington's disease and related disorders |
Family Cites Families (66)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6582908B2 (en) | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
US5700922A (en) | 1991-12-24 | 1997-12-23 | Isis Pharmaceuticals, Inc. | PNA-DNA-PNA chimeric macromolecules |
CA2116280A1 (en) * | 1993-03-05 | 1994-09-06 | Marcy E. Macdonald | Huntingtin dna, protein and uses thereof |
FR2705099B1 (fr) | 1993-05-12 | 1995-08-04 | Centre Nat Rech Scient | Oligonucléotides phosphorothioates triesters et procédé de préparation. |
US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
US5595756A (en) | 1993-12-22 | 1997-01-21 | Inex Pharmaceuticals Corporation | Liposomal compositions for enhanced retention of bioactive agents |
US20040266706A1 (en) | 2002-11-05 | 2004-12-30 | Muthiah Manoharan | Cross-linked oligomeric compounds and their use in gene modulation |
ES2192672T3 (es) | 1996-11-18 | 2003-10-16 | Takeshi Imanishi | Nuevos analogos de nucleotidos. |
NZ503765A (en) | 1997-09-12 | 2002-04-26 | Exiqon As | Bi-cyclic and tri-cyclic nucleotide analogues |
US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
US20010049436A1 (en) * | 1998-04-01 | 2001-12-06 | Wen-Qiang Zhou | Mixed-backbone oligonucleotides containing pops blocks to obtain reduced phosphorothioate content |
US20030228597A1 (en) | 1998-04-13 | 2003-12-11 | Cowsert Lex M. | Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation |
WO1999060012A1 (en) * | 1998-05-21 | 1999-11-25 | Isis Pharmaceuticals, Inc. | Compositions and methods for non-parenteral delivery of oligonucleotides |
US6277967B1 (en) * | 1998-07-14 | 2001-08-21 | Isis Pharmaceuticals, Inc. | Carbohydrate or 2′-modified oligonucleotides having alternating internucleoside linkages |
US5998148A (en) | 1999-04-08 | 1999-12-07 | Isis Pharmaceuticals Inc. | Antisense modulation of microtubule-associated protein 4 expression |
US6147200A (en) | 1999-08-19 | 2000-11-14 | Isis Pharmaceuticals, Inc. | 2'-O-acetamido modified monomers and oligomers |
WO2001079283A1 (en) | 2000-04-13 | 2001-10-25 | University Of British Columbia | Modulating cell survival by modulating huntingtin function |
AU2001296412A1 (en) | 2000-09-29 | 2002-04-08 | Isis Pharmaceuticals, Inc. | Antisense modulation of mekk4 expression |
US20050191638A1 (en) | 2002-02-20 | 2005-09-01 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
CA2452458A1 (en) | 2001-07-03 | 2003-01-16 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
DK1408964T3 (da) * | 2001-07-26 | 2007-05-21 | Merck Patent Gmbh | Hidtil ukendt anvendelse af 2-5-(4-fluorphenyl)-3-pyridylmethylaminomethyl-chroman og fysiologisk acceptable salte deraf |
AU2002326589B2 (en) | 2001-08-07 | 2008-06-05 | University Of Delaware | Compositions and methods for the prevention and treatment of Huntington's disease |
US20040096880A1 (en) | 2001-08-07 | 2004-05-20 | Kmiec Eric B. | Compositions and methods for the treatment of diseases exhibiting protein misassembly and aggregation |
US7176303B2 (en) | 2003-11-06 | 2007-02-13 | Isis Pharmaceuticals, Inc. | Modulation of STAT5 expression |
EP2221377B2 (en) | 2002-02-01 | 2017-05-17 | Life Technologies Corporation | Oligonucleotide compositions with enhanced efficiency |
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) |
US20040092465A1 (en) | 2002-11-11 | 2004-05-13 | Isis Pharmaceuticals Inc. | Modulation of huntingtin interacting protein 1 expression |
US20040102398A1 (en) | 2002-11-23 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of B7H expression |
US20050106731A1 (en) | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
US20050255086A1 (en) | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
US20040137471A1 (en) | 2002-09-18 | 2004-07-15 | Timothy Vickers | Efficient reduction of target RNA's by single-and double-stranded oligomeric compounds |
CA2504694C (en) | 2002-11-05 | 2013-10-01 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
US7250496B2 (en) | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
AU2003295600A1 (en) | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
AU2004239114B2 (en) | 2003-05-14 | 2008-03-13 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
ATE555118T1 (de) | 2003-08-28 | 2012-05-15 | Takeshi Imanishi | Neue synthetische nukleidsäuren vom typ mit quervernetzter n-o-bindung |
WO2005027980A1 (en) | 2003-09-12 | 2005-03-31 | University Of Massachusetts | Rna interference for the treatment of gain-of-function disorders |
CA2538252C (en) | 2003-09-18 | 2014-02-25 | Isis Pharmaceuticals, Inc. | 4'-thionucleosides and oligomeric compounds |
WO2005045032A2 (en) | 2003-10-20 | 2005-05-19 | Sima Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF EARLY GROWTH RESPONSE GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
US20050256072A1 (en) | 2004-02-09 | 2005-11-17 | University Of Massachusetts | Dual functional oligonucleotides for use in repressing mutant gene expression |
WO2005083436A1 (en) | 2004-02-26 | 2005-09-09 | Bayer Healthcare Ag | Diagnostics and therapeutics for diseases associated with integrin-linked kinase 1 (ilk1) |
EP1735443A2 (en) | 2004-04-14 | 2006-12-27 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
US7374927B2 (en) | 2004-05-03 | 2008-05-20 | Affymetrix, Inc. | Methods of analysis of degraded nucleic acid samples |
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 |
US7323308B2 (en) | 2004-09-03 | 2008-01-29 | Affymetrix, Inc. | Methods of genetic analysis of E. coli |
JP4917549B2 (ja) | 2005-01-14 | 2012-04-18 | ダウ グローバル テクノロジーズ エルエルシー | チタノシリケートの再生、及び活性な酸化触媒の再構成 |
EP1891217A2 (en) | 2005-05-27 | 2008-02-27 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of stromal cell-derived factor-1 (sdf-1) gene expression using short interfering nucleic acid (sina) |
US20070161591A1 (en) | 2005-08-18 | 2007-07-12 | University Of Massachusetts | Methods and compositions for treating neurological disease |
JP5111385B2 (ja) * | 2005-10-28 | 2013-01-09 | アルナイラム ファーマシューティカルズ, インコーポレイテッド | ハンチンチン遺伝子の発現を抑制するための組成物および方法 |
EP1976567B1 (en) * | 2005-12-28 | 2020-05-13 | The Scripps Research Institute | Natural antisense and non-coding rna transcripts as drug targets |
US7951934B2 (en) | 2006-01-26 | 2011-05-31 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to huntingtin |
CA2640171C (en) | 2006-01-27 | 2014-10-28 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
GB0605337D0 (en) * | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
US8076306B2 (en) | 2006-04-12 | 2011-12-13 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to hepcidin |
WO2007134181A2 (en) | 2006-05-11 | 2007-11-22 | Isis Pharmaceuticals, Inc. | 5'-modified bicyclic nucleic acid analogs |
CA2662704A1 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
US20080039415A1 (en) | 2006-08-11 | 2008-02-14 | Gregory Robert Stewart | Retrograde transport of sirna and therapeutic uses to treat neurologic disorders |
CA2660523C (en) | 2006-08-11 | 2019-03-19 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
US20080068922A1 (en) | 2006-09-12 | 2008-03-20 | Voss Klaus-W | Device for blending a binder component and a hardener component for producing a ready-made filler |
JP2008068470A (ja) | 2006-09-13 | 2008-03-27 | Kyocera Mita Corp | 電子機器、ジョブデータ認証受信プログラム |
EP2125852B1 (en) | 2007-02-15 | 2016-04-06 | Ionis Pharmaceuticals, Inc. | 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom |
CN101790385A (zh) * | 2007-07-12 | 2010-07-28 | 普罗森那技术公司 | 用于使化合物靶向多种选定器官、组织或肿瘤细胞的分子 |
CN101821277B (zh) | 2007-08-15 | 2014-05-07 | Isis制药公司 | 四氢吡喃核酸类似物 |
WO2011097388A1 (en) | 2010-02-03 | 2011-08-11 | Alnylam Pharmaceuticals, Inc. | Selective inhibition of polyglutamine protein expression |
-
2010
- 2010-09-10 SI SI201031404A patent/SI2475675T1/sl unknown
- 2010-09-10 MX MX2012003006A patent/MX340397B/es active IP Right Grant
- 2010-09-10 PL PL10816206T patent/PL2475675T3/pl unknown
- 2010-09-10 EP EP16197941.4A patent/EP3178933B1/en active Active
- 2010-09-10 RU RU2012114198/10A patent/RU2562861C2/ru active
- 2010-09-10 EP EP19199744.4A patent/EP3626823A1/en active Pending
- 2010-09-10 KR KR1020127009137A patent/KR101774526B1/ko active IP Right Grant
- 2010-09-10 AU AU2010292003A patent/AU2010292003B2/en active Active
- 2010-09-10 NZ NZ714887A patent/NZ714887A/en unknown
- 2010-09-10 EP EP10816206.6A patent/EP2475675B1/en active Active
- 2010-09-10 DK DK10816206.6T patent/DK2475675T3/en active
- 2010-09-10 US US13/395,188 patent/US8906873B2/en active Active
- 2010-09-10 KR KR1020207031044A patent/KR102279458B1/ko active IP Right Grant
- 2010-09-10 KR KR1020187037590A patent/KR102175230B1/ko active IP Right Grant
- 2010-09-10 RU RU2015132208A patent/RU2751847C9/ru active
- 2010-09-10 PT PT108162066T patent/PT2475675T/pt unknown
- 2010-09-10 JP JP2012528949A patent/JP5809146B2/ja active Active
- 2010-09-10 HU HUE10816206A patent/HUE033113T2/en unknown
- 2010-09-10 CN CN202010532625.8A patent/CN111705058A/zh active Pending
- 2010-09-10 KR KR1020197026749A patent/KR102173836B1/ko active Application Filing
- 2010-09-10 CA CA2773886A patent/CA2773886C/en active Active
- 2010-09-10 BR BR112012005448-1A patent/BR112012005448B1/pt active IP Right Grant
- 2010-09-10 KR KR1020177024113A patent/KR20170102069A/ko active Application Filing
- 2010-09-10 CN CN201510262406.1A patent/CN104894129B/zh active Active
- 2010-09-10 WO PCT/US2010/048532 patent/WO2011032045A1/en active Application Filing
- 2010-09-10 NZ NZ599032A patent/NZ599032A/en unknown
- 2010-09-10 ES ES10816206.6T patent/ES2615534T3/es active Active
- 2010-09-10 NZ NZ624783A patent/NZ624783A/en unknown
- 2010-09-10 CN CN201080051393.9A patent/CN102625809B/zh active Active
- 2010-09-10 CA CA2931725A patent/CA2931725C/en active Active
- 2010-09-10 ES ES16197941T patent/ES2772825T3/es active Active
-
2012
- 2012-03-05 IL IL218482A patent/IL218482A/en active IP Right Grant
- 2012-03-09 MX MX2016004031A patent/MX353152B/es unknown
-
2014
- 2014-10-30 US US14/528,656 patent/US9273315B2/en active Active
-
2015
- 2015-09-10 JP JP2015178281A patent/JP6153578B2/ja active Active
-
2016
- 2016-01-25 US US15/005,712 patent/US9683236B2/en active Active
- 2016-03-04 AU AU2016201431A patent/AU2016201431A1/en not_active Abandoned
-
2017
- 2017-02-15 IL IL250611A patent/IL250611B/en active IP Right Grant
- 2017-05-16 US US15/596,249 patent/US10202603B2/en active Active
- 2017-05-30 JP JP2017106418A patent/JP2017184754A/ja active Pending
- 2017-12-14 AU AU2017276286A patent/AU2017276286B2/en active Active
-
2018
- 2018-09-26 JP JP2018180351A patent/JP6704439B2/ja active Active
-
2019
- 2019-02-08 US US16/270,983 patent/US10619158B2/en active Active
- 2019-07-24 IL IL268246A patent/IL268246B/en active IP Right Grant
- 2019-12-30 AU AU2019284142A patent/AU2019284142B2/en active Active
-
2020
- 2020-02-26 US US16/801,431 patent/US10837016B2/en active Active
- 2020-03-03 IL IL273017A patent/IL273017B/en unknown
- 2020-05-12 JP JP2020083833A patent/JP2020115894A/ja active Pending
- 2020-10-12 US US17/068,185 patent/US11421231B2/en active Active
-
2022
- 2022-07-18 US US17/813,249 patent/US20230023015A1/en active Pending
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
MX353152B (es) | Modulación de expresión de huntingtina. | |
MX2012012624A (es) | Modulacion de la expresion de transtiretina. | |
MX350944B (es) | Modulación antisentido de la expresión de gccr. | |
EP3489360A3 (en) | Modulation of nuclear-retained rna | |
HK1249446A1 (zh) | 用於治療乳糜瀉的組合物和方法 | |
MX353322B (es) | Modulacion antisentido de la expresion de gcgr. | |
IL259413B (en) | Preparations for the treatment of Gaucher disease | |
MY163275A (en) | Ibat inhibitors for the treatment of liver diseases | |
MX2010004219A (es) | Agentes de enlace de cd19 y usos de los mismos. | |
PH12015501470A1 (en) | Compositions and methods for the control of nematodes and soil borne diseases | |
BR112012003092A2 (pt) | composições e métodos para implantação de tecido adiposo processado e produtos de tecido adiposo processados. | |
PL2349231T3 (pl) | Kompozycje i metody leczenia chorób kopyta | |
WO2012174476A3 (en) | Antisense modulation of fibroblast growth factor receptor 4 expression | |
EA201000559A1 (ru) | Композиции и способы применения антител к склеростину | |
BRPI1009920A2 (pt) | composições bacterianas para profilaxina e tratamento de doença degenerativa. | |
TN2009000521A1 (en) | Rnai inhibition of alpha-enac expression | |
BRPI0908087A2 (pt) | Composições e métodos para implante de tecido adipose e produtos de tecido adiposo | |
GB0920703D0 (en) | Compositions containing satiogens and methods of use | |
ZA201101213B (en) | Compositions and methods for treating disorders associated with overweight animals | |
JOP20180077A1 (ar) | تركيبات وطرق لحمض صفراوي تخليقي | |
CR11870A (es) | Composiciones de perácido y 2-hidroxi ácido orgánico y métodos para tratar productos agrícolas | |
BRPI1009045A2 (pt) | arco ortodôntico de baixa força que tem blocos de engate para tratamento aprimorado | |
HK1245679A1 (zh) | 用於治療皮膚疾病和病症的方法和組合物 | |
IL196920A0 (en) | Compositions and methods using anti-csi antibodies to treat multiple myeloma | |
EP2271352A4 (en) | COMPOSITIONS AND METHODS FOR TREATING NEOPLASTIC DISEASE |
Legal Events
Date | Code | Title | Description |
---|---|---|---|
HC | Change of company name or juridical status |
Owner name: SHOREWOOD INTELLECTUAL PROPERTY HOLDINGS B.V. |
|
FG | Grant or registration |