DK2850186T3 - Sammensætninger og fremgangsmåder til at modulere smn-genfamilie-ekspression - Google Patents
Sammensætninger og fremgangsmåder til at modulere smn-genfamilie-ekspression Download PDFInfo
- Publication number
- DK2850186T3 DK2850186T3 DK13790819.0T DK13790819T DK2850186T3 DK 2850186 T3 DK2850186 T3 DK 2850186T3 DK 13790819 T DK13790819 T DK 13790819T DK 2850186 T3 DK2850186 T3 DK 2850186T3
- Authority
- DK
- Denmark
- Prior art keywords
- oligonucleotide
- stranded oligonucleotide
- smn2
- nucleotides
- sequence
- 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
-
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- 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
-
- 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
- C12N15/1131—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 against viruses
-
- 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
- C12N15/1131—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 against viruses
- C12N15/1132—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 against viruses against retroviridae, e.g. HIV
-
- 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
- C12N2310/323—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)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Pain & Pain Management (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- AIDS & HIV (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Claims (19)
1. Enkeltstrenget oligonukleotid med en sekvens 5'-X-Y-Z, hvor X er et hvilket som helst nukleotid, Y er en nukleotidsekvens af 6 nukleotiders længde, der ikke er en seed-sekvens af et humant mikroRNA, og Z er en nukleotidsekvens af 1-23 nukleotiders længde, hvor det enkeltstrengede oligonukleotid er komplementært med mindst 8 på hinanden følgende nukleotider af en PRC2-associeret region af et SMN-gen, og hvor det enkeltstrengede oligonukleotid aktiverer eller forstærker ekspression af SMN1 eller SMN2 ved at mindske eller forhindre PRC2-medieret undertrykkelse af SMN1 eller SMN2.
2. Enkeltstrenget oligonukleotid ifølge krav 1, hvor (a) oligonukleotidet ikke omfatter fire eller flere på hinanden følgende guanosinnukleotider, eventuelt hvor oligonukleotidet ikke omfatter tre eller flere på hinanden følgende guanosinnukleotider; og/eller (b) oligonukleotidet i. er af 8 til 30 nukleotiders længde, eller ii. er af 8 til 10 nukleotiders længde og alle undtagen 1, 2, eller 3 af nukleotiderne af den komplementære sekvens af den PRC2-associerede region er cytosin- eller guanosinnukleotider.
3. Enkeltstrenget oligonukleotid ifølge et hvilket som helst af de foregående krav, hvor mindst et nukleotid af oligonukleotidet er en nukleotidanalog, hvor eventuelt den mindst ene nukleotidanalog resulterer i en forøgelse af Tm af oligonukleotidet i området fra 1 til 5 °C sammenlignet med et oligonukleotid, der ikke har den mindst ene nukleotidanalog.
4. Enkeltstrenget oligonukleotid ifølge et hvilket som helst af kravene 1 til 3, hvor (a) mindst et nukleotid af oligonukleotidet omfatter en 2' O-methyl, hvor eventuelt (b) hvert nukleotid af oligonukleotidet omfatter en 2' O-methyl.
5. Enkeltstrenget oligonukleotid ifølge et hvilket som helst af kravene 1 til 3 eller 4(a), hvor (a) oligonukleotidet omfatter mindst et ribonukleotid, mindst et deoxyribonukleotid, eller mindst et broforbundet ("bridged") nukleotid, hvor eventuelt det broforbundne nukleotid er et LNA-nukleotid, et cEt-nukleotid eller et ENA-modificeret nukleotid; eller (b) nukleotiderne af oligonukleotidet omfatter deoxyribonukleotider flankeret af mindst et LNA-nukleotid ved hver af 5'- og 3'-enderne af deoxyribonukleotiderne.
6. Enkeltstrenget oligonukleotid ifølge et hvilket som helst af kravene 1 til 3, hvor (a) hvert nukleotid af oligonukleotidet er et LNA-nukleotid; eller (b) nukleotiderne af oligonukleotidet omfatter (i) skiftevis deoxyribonukleotider og 2'-fluor-deoxyribonukleotider; (ii) skiftevis deoxyribonukleotider og 2'-O-methylnukleotider; (iii) skiftevis deoxyribonukleotider og ENA-nukleotidanaloger; eller (iv) skiftevis deoxyribonukleotider og LNA-nukleotider; eller (v) skiftevis LNA-nukleotider og 2'-O-methylnukleotider.
7. Enkeltstrenget oligonukleotid ifølge krav 6(b), hvor 5'-nukleotidet af oligonukleotidet er et deoxyribonukleotid eller et LNA-nukleotid.
8. Enkeltstrenget oligonukleotid ifølge et hvilket som helst af kravene 1 til 7, endvidere omfattende phosphorthioat-internukleotidkoblinger mellem mindst to nukleotider, eventuelt endvidere omfattende phosphorthioat- internukleotidkoblinger mellem alle nukleotider.
9. Enkeltstrenget oligonukleotid ifølge et hvilket som helst af kravene 1 til 8, hvor (a) nukleotidet i 3'-positionen af oligonukleotidet haren 3'-hydroxylgruppe; (b) nukleotidet i 3'-positionen af oligonukleotidet haren 3'-thiophosphat; og/eller (c) oligonukleotidet endvidere omfatter en biotindel konjugeret til 5'-nukleotidet.
10. Enkeltstrenget oligonukleotid omfattende en komplementaritetsregion der er komplementær med mindst 8 på hinanden følgende nukleotider af en PRC2-associeret region af et SMN-gen, hvor oligonukleotidet aktiverer eller forstærker ekspression af SMN1 eller SMN2 ved at mindske eller forhindre PRC2-medieret undertrykkelse af SMN1 eller SMN2, og hvor oligonukleotidet har mindst en af: a) en sekvens der er 5'X-Y-Z, hvor X er et hvilket som helst nukleotid, og hvor X er forankret ved 5'-enden af oligonukleotidet, Y er en nukleotidsekvens af 6 nukleotiders længde, der ikke er en human seed-sekvens af et mikroRNA, og Z er en nukleotidsekvens af 1 til 23 nukleotiders længde; b) en sekvens der ikke omfatter tre eller flere på hinanden følgende guanosinnukleotider; c) en sekvens der har mindre end et tærkselniveau af sekvensidentitet med hver sekvens af nukleotider, med samme længde, der er mellem 50 kilobaser opstrøms fra en 5'-ende af et ikke-målgen og 50 kilobaser nedstrøms fra en 3'-ende af ikke-målgenet; d) en sekvens der er komplementær med en PRC2-associeret region der koder for et RNA, der danner en sekundær struktur omfattende mindst to enkeltstrengede sløjfer; og/eller e) en sekvens der har mere end 60% G-C-indhold; hvor eventuelt i et hvilket som helst af (a)-(e) oligonukleotidet har sekvensen 5'X-Y-Z, og oligonukleotidet er af 8-50 nukleotiders længde.
11. Sammensætning omfattende et enkeltstrenget oligonukleotid ifølge et hvilket som helst af kravene 1 til 10 og en bærer, hvor eventuelt (a) sammensætningen er en farmaceutisk sammensætning omfattende en farmaceutisk acceptabel bærer; (b) sammensætningen omfatter det enkeltstrengede oligonukleotid i en bufferopløsning, og/eller (c) oligonukleotidet er konjugeret til bæreren, hvor eventuelt bæreren er et peptid eller et steroid.
12. Kit omfattende en beholder, der indeholder sammensætningen ifølge krav 11.
13. Fremgangsmåde til forøgelse af ekspression af SMN1 eller SMN2 i en celle, hvilken fremgangsmåde ikke er en fremgangsmåde til behandling af menneskeeller dyrekroppen med kirurgi eller terapi, hvor fremgangsmåden omfatter levering af det enkeltstrengede oligonukleotid ifølge et hvilket som helst af kravene 1 til 10 ind i cellen, hvor eventuelt levering af det enkeltstrengede oligonukleotid ind i cellen resulterer i et niveau af ekspression af SMN1 eller SMN2, der er mindst 50% større end et niveau af ekspression af SMN1 eller SMN2 i en kontrolcelle, der ikke omfatter det enkeltstrengede oligonukleotid.
14. Enkeltstrenget oligonukleotid som defineret i et hvilket som helst af kravene 1-10 til anvendelse i en fremgangsmåde til forøgelse af niveauer af SMN1 eller SMN2 i et individ, hvor fremgangsmåden omfatter indgivelse af det enkeltstrengede oligonukleotid til individet.
15. Enkeltstrenget oligonukleotid som defineret i et hvilket som helst af kravene 1-10 til anvendelse i en fremgangsmåde til behandling afen tilstand associeret med reducerede niveauer af SMN1 eller SMN2 i et individ, hvor fremgangsmåden omfatter indgivelse af det enkeltstrengede oligonukleotid til individet, hvor eventuelt tilstanden er spinal muskelatrofi.
16. In vitro fremgangsmåde til forøgelse af ekspression af SMN-protein i en celle, hvor fremgangsmåden omfatter: levering til cellen af et første enkeltstrenget oligonukleotid komplementært med mindst 8 på hinanden følgende nukleotider af en PRC2-associeret region af SMN2 og et andet enkeltstrenget oligonukleotid komplementært med en splejsningskontrolsekvens af en mRNA-precursor af SMN2, i mængder der er tilstrækkelige til at forøge ekspression af et modnet mRNA af SMN2, der omfatter exon 7 i cellen, hvor det første enkeltstrengede oligonukleotid aktiverer eller forstærker ekspression af SMN1 eller SMN2 ved at mindske eller forhindre PRC2-medieret undertrykkelse af SMN1 eller SMN2, og hvor eventuelt det første enkeltstrengede oligonukleotid er kovalent bundet til det andet enkeltstrengede oligonukleotid gennem en linker.
17. Et første enkeltstrenget oligonukleotid komplementært med mindst 8 på hinanden følgende nukleotider af en PRC2-associeret region af SMN2, hvor det første enkeltstrengede oligonukleotid aktiverer eller forstærker ekspression af SMN1 eller SMN2 ved at mindske eller forhindre PRC2-medieret undertrykkelse af SMN1 eller SMN2, og et andet enkeltstrenget oligonukleotid komplementært med en splejsningskontrolsekvens af en mRNA-precursor af SMN2 til anvendelse i en fremgangsmåde til behandling af spinal muskelatrofi i et individ, hvor fremgangsmåden omfatter: indgivelse til individet af nævnte første og anden enkeltstrengede oligonukleotider i mængder, der er tilstrækkelige til at øge ekspression af SMN-protein i individet.
18. Sammensætning omfattende: et første enkeltstrenget oligonukleotid komplementært med mindst 8 på hinanden følgende nukleotider af en PRC2-associeret region af SMN2, og et andet enkeltstrenget oligonukleotid komplementært med en splejsningskontrolsekvens af en mRNA-precursor af SMN2, hvor det første enkeltstrengede oligonukleotid aktiverer eller forstærker ekspression af SMN1 eller SMN2 ved at mindske eller forhindre PRC2-medieret undertrykkelse af SMN1 eller SMN2, og hvor eventuelt det første enkeltstrengede oligonukleotid er kovalent bundet til det andet enkeltstrengede oligonukleotid gennem en linker.
19. Forbindelse omfattende den generelle formel A-B-C, hvor A er et enkeltstrenget oligonukleotid komplementært med mindst 8 på hinanden følgende nukleotider af en PRC2-associeret region af et gen, hvor det enkeltstrengede oligonukleotid aktiverer eller forstærker ekspression af SMN1 eller SMN2 ved at mindske eller forhindre PRC2-medieret undertrykkelse af SMN1 eller SMN2, B er en linker, og C er et enkeltstrenget oligonukleotid komplementært med en splejsningskontrolsekvens af en mRNA-precursor af genet, hvor genet eventuelt er SMN2.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261647858P | 2012-05-16 | 2012-05-16 | |
US201261719394P | 2012-10-27 | 2012-10-27 | |
US201361785529P | 2013-03-14 | 2013-03-14 | |
PCT/US2013/041440 WO2013173638A1 (en) | 2012-05-16 | 2013-05-16 | Compositions and methods for modulating smn gene family expression |
Publications (1)
Publication Number | Publication Date |
---|---|
DK2850186T3 true DK2850186T3 (da) | 2019-04-08 |
Family
ID=49584305
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK13790819.0T DK2850186T3 (da) | 2012-05-16 | 2013-05-16 | Sammensætninger og fremgangsmåder til at modulere smn-genfamilie-ekspression |
Country Status (13)
Country | Link |
---|---|
US (1) | US10059941B2 (da) |
EP (1) | EP2850186B1 (da) |
JP (1) | JP2015523854A (da) |
KR (1) | KR20150030205A (da) |
CN (1) | CN104540947A (da) |
AU (1) | AU2013262649A1 (da) |
CA (1) | CA2873794A1 (da) |
DK (1) | DK2850186T3 (da) |
EA (1) | EA201492123A1 (da) |
HK (1) | HK1208700A1 (da) |
SG (1) | SG11201407483YA (da) |
WO (1) | WO2013173638A1 (da) |
ZA (1) | ZA201409228B (da) |
Families Citing this family (37)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2011325956B2 (en) | 2010-11-12 | 2016-07-14 | 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 |
AP2014008100A0 (en) | 2012-05-16 | 2014-12-31 | Gen Hospital Corp | Compositions and methods for modulating hemoglobingene family expression |
CA2873766A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating atp2a2 expression |
EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
EP2850185A4 (en) | 2012-05-16 | 2015-12-30 | Rana Therapeutics Inc | COMPOSITIONS AND METHODS FOR MODULATING UTRN EXPRESSION |
US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | 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 |
EP3560502A1 (en) | 2013-04-12 | 2019-10-30 | The Curators of the University of Missouri | Smn2 element 1 antisense compositions and methods and uses thereof |
US10174328B2 (en) | 2013-10-04 | 2019-01-08 | Translate Bio Ma, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
CN103911346B (zh) * | 2014-03-27 | 2016-09-07 | 江苏雄鸣医药科技有限公司 | 一种用于治疗脊髓性肌萎缩症药物筛选的细胞模型 |
US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
CA2966044A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
EP3271460A4 (en) | 2015-03-17 | 2019-03-13 | The General Hospital Corporation | INTERACTOME RNA OF COMPLEX REPRESSIVE POLYCOMB 1 (PRC1) |
WO2016164896A2 (en) | 2015-04-10 | 2016-10-13 | Ionis Pharmaceuticals, Inc. | Modulation of smn expression |
US11249071B2 (en) | 2015-04-24 | 2022-02-15 | California Institute Of Technology | Reactivation of x chromosome genes |
CA2996164A1 (en) | 2015-08-28 | 2017-03-09 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
WO2017075030A1 (en) * | 2015-10-26 | 2017-05-04 | Rana Therapeutics, Inc. | Methods and compositions for increasing smn expression |
CA3005434A1 (en) | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
WO2017218884A1 (en) * | 2016-06-16 | 2017-12-21 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of smn expression |
WO2018081661A1 (en) | 2016-10-27 | 2018-05-03 | California Institute Of Technology | Hdac inhibitor compositions for reactivation of the x chromosome |
WO2018085198A1 (en) | 2016-11-01 | 2018-05-11 | The Research Foundation For The State University Of New York | 5-halouracil-modified micrornas and their use in the treatment of cancer |
US20180193270A1 (en) | 2016-11-29 | 2018-07-12 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
WO2018226788A1 (en) * | 2017-06-07 | 2018-12-13 | University Of Massachusetts | Anti-adam33 oligonucleotides and related methods |
US20200208152A1 (en) | 2017-09-08 | 2020-07-02 | Mina Therapeutics Limited | Stabilized sarna compositions and methods of use |
EP3679138B1 (en) | 2017-09-08 | 2023-03-22 | MiNA Therapeutics Limited | Hnf4a sarna compositions and methods of use |
SG11202001783YA (en) * | 2017-10-12 | 2020-03-30 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
WO2020069109A1 (en) | 2018-09-26 | 2020-04-02 | Greenlight Biosciences, Inc. | Control of coleopteran insects |
EP3877530A1 (en) | 2018-11-08 | 2021-09-15 | Greenlight Biosciences, Inc. | Control of insect infestation |
EP3908671A4 (en) * | 2019-01-09 | 2022-10-05 | Coyote Bioscience USA Inc. | METHODS AND SYSTEMS FOR IDENTIFYING SPINAL MUSD ATROPHY |
US20220280548A1 (en) * | 2019-08-15 | 2022-09-08 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
JP2022545101A (ja) | 2019-08-19 | 2022-10-25 | ミナ セラピューティクス リミテッド | オリゴヌクレオチドコンジュゲート組成物および使用方法 |
KR20220148230A (ko) | 2020-02-28 | 2022-11-04 | 아이오니스 파마수티컬즈, 인코포레이티드 | Smn2를 조절하기 위한 화합물 및 방법 |
CN113444722A (zh) * | 2020-03-24 | 2021-09-28 | 中国科学院脑科学与智能技术卓越创新中心 | 单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用 |
WO2022178273A1 (en) * | 2021-02-19 | 2022-08-25 | University Of Florida Research Foundation, Incorporated | Methods and compositions to confer regulation to gene therapy cargoes by heterologous use of alternative splicing cassettes |
WO2023020421A1 (en) * | 2021-08-16 | 2023-02-23 | Hangzhou Jiayin Biotech Ltd. | Regulation of imprinting silenced genes by smn1 and snrpn expression and uses thereof |
CA3230274A1 (en) * | 2021-09-03 | 2023-03-09 | Alfica Sehgal | Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas |
Family Cites Families (400)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
US4469863A (en) | 1980-11-12 | 1984-09-04 | Ts O Paul O P | Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof |
US5023243A (en) | 1981-10-23 | 1991-06-11 | Molecular Biosystems, Inc. | Oligonucleotide therapeutic agent and method of making same |
JP2547714B2 (ja) | 1981-10-23 | 1996-10-23 | モルキユラ− バイオシステムズ インコ−ポレテツド | オリゴヌクレオチド治療剤及びその製法 |
US4476301A (en) | 1982-04-29 | 1984-10-09 | Centre National De La Recherche Scientifique | Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon |
JPS5927900A (ja) | 1982-08-09 | 1984-02-14 | Wakunaga Seiyaku Kk | 固定化オリゴヌクレオチド |
FR2540122B1 (fr) | 1983-01-27 | 1985-11-29 | Centre Nat Rech Scient | Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application |
US4605735A (en) | 1983-02-14 | 1986-08-12 | Wakunaga Seiyaku Kabushiki Kaisha | Oligonucleotide derivatives |
US4948882A (en) | 1983-02-22 | 1990-08-14 | Syngene, Inc. | Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis |
US4824941A (en) | 1983-03-10 | 1989-04-25 | Julian Gordon | Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems |
DE3329892A1 (de) | 1983-08-18 | 1985-03-07 | Köster, Hubert, Prof. Dr., 2000 Hamburg | Verfahren zur herstellung von oligonucleotiden |
US4587044A (en) | 1983-09-01 | 1986-05-06 | The Johns Hopkins University | Linkage of proteins to nucleic acids |
US5118802A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside |
US5550111A (en) | 1984-07-11 | 1996-08-27 | Temple University-Of The Commonwealth System Of Higher Education | Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof |
US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
US5258506A (en) | 1984-10-16 | 1993-11-02 | Chiron Corporation | Photolabile reagents for incorporation into oligonucleotide chains |
US5430136A (en) | 1984-10-16 | 1995-07-04 | Chiron Corporation | Oligonucleotides having selectably cleavable and/or abasic sites |
US4828979A (en) | 1984-11-08 | 1989-05-09 | Life Technologies, Inc. | Nucleotide analogs for nucleic acid labeling and detection |
FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
US5405938A (en) | 1989-12-20 | 1995-04-11 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
US5235033A (en) | 1985-03-15 | 1993-08-10 | Anti-Gene Development Group | Alpha-morpholino ribonucleoside derivatives and polymers thereof |
US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
US4762779A (en) | 1985-06-13 | 1988-08-09 | Amgen Inc. | Compositions and methods for functionalizing nucleic acids |
US5317098A (en) | 1986-03-17 | 1994-05-31 | Hiroaki Shizuya | Non-radioisotope tagging of fragments |
JPS638396A (ja) | 1986-06-30 | 1988-01-14 | Wakunaga Pharmaceut Co Ltd | ポリ標識化オリゴヌクレオチド誘導体 |
DE3788914T2 (de) | 1986-09-08 | 1994-08-25 | Ajinomoto Kk | Verbindungen zur Spaltung von RNS an eine spezifische Position, Oligomere, verwendet bei der Herstellung dieser Verbindungen und Ausgangsprodukte für die Synthese dieser Oligomere. |
US5264423A (en) | 1987-03-25 | 1993-11-23 | The United States Of America As Represented By The Department Of Health And Human Services | Inhibitors for replication of retroviruses and for the expression of oncogene products |
US5276019A (en) | 1987-03-25 | 1994-01-04 | The United States Of America As Represented By The Department Of Health And Human Services | Inhibitors for replication of retroviruses and for the expression of oncogene products |
US4904582A (en) | 1987-06-11 | 1990-02-27 | Synthetic Genetics | Novel amphiphilic nucleic acid conjugates |
ATE113059T1 (de) | 1987-06-24 | 1994-11-15 | Florey Howard Inst | Nukleosid-derivate. |
US5585481A (en) | 1987-09-21 | 1996-12-17 | Gen-Probe Incorporated | Linking reagents for nucleotide probes |
US4924624A (en) | 1987-10-22 | 1990-05-15 | Temple University-Of The Commonwealth System Of Higher Education | 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof |
US5188897A (en) | 1987-10-22 | 1993-02-23 | Temple University Of The Commonwealth System Of Higher Education | Encapsulated 2',5'-phosphorothioate oligoadenylates |
US5525465A (en) | 1987-10-28 | 1996-06-11 | Howard Florey Institute Of Experimental Physiology And Medicine | Oligonucleotide-polyamide conjugates and methods of production and applications of the same |
DE3738460A1 (de) | 1987-11-12 | 1989-05-24 | Max Planck Gesellschaft | Modifizierte oligonukleotide |
DE3855864T2 (de) | 1987-11-30 | 1997-09-25 | Univ Iowa Res Found | Durch modifikationen an der 3'-terminalen phosphodiesterbindung stabilisierte dna moleküle, ihre verwendung als nukleinsäuresonden sowie als therapeutische mittel zur hemmung der expression spezifischer zielgene |
US5403711A (en) | 1987-11-30 | 1995-04-04 | University Of Iowa Research Foundation | Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved |
US5082830A (en) | 1988-02-26 | 1992-01-21 | Enzo Biochem, Inc. | End labeled nucleotide probe |
EP0406309A4 (en) | 1988-03-25 | 1992-08-19 | The University Of Virginia Alumni Patents Foundation | Oligonucleotide n-alkylphosphoramidates |
US5278302A (en) | 1988-05-26 | 1994-01-11 | University Patents, Inc. | Polynucleotide phosphorodithioates |
US5109124A (en) | 1988-06-01 | 1992-04-28 | Biogen, Inc. | Nucleic acid probe linked to a label having a terminal cysteine |
US5216141A (en) | 1988-06-06 | 1993-06-01 | Benner Steven A | Oligonucleotide analogs containing sulfur linkages |
US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
US5262536A (en) | 1988-09-15 | 1993-11-16 | E. I. Du Pont De Nemours And Company | Reagents for the preparation of 5'-tagged oligonucleotides |
US5512439A (en) | 1988-11-21 | 1996-04-30 | Dynal As | Oligonucleotide-linked magnetic particles and uses thereof |
US5599923A (en) | 1989-03-06 | 1997-02-04 | Board Of Regents, University Of Tx | Texaphyrin metal complexes having improved functionalization |
US5457183A (en) | 1989-03-06 | 1995-10-10 | Board Of Regents, The University Of Texas System | Hydroxylated texaphyrins |
US5391723A (en) | 1989-05-31 | 1995-02-21 | Neorx Corporation | Oligonucleotide conjugates |
US5256775A (en) | 1989-06-05 | 1993-10-26 | Gilead Sciences, Inc. | Exonuclease-resistant oligonucleotides |
US4958013A (en) | 1989-06-06 | 1990-09-18 | Northwestern University | Cholesteryl modified oligonucleotides |
US5451463A (en) | 1989-08-28 | 1995-09-19 | Clontech Laboratories, Inc. | Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides |
US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
US5254469A (en) | 1989-09-12 | 1993-10-19 | Eastman Kodak Company | Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures |
US5399676A (en) | 1989-10-23 | 1995-03-21 | Gilead Sciences | Oligonucleotides with inverted polarity |
US5264564A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences | Oligonucleotide analogs with novel linkages |
US5264562A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences, Inc. | Oligonucleotide analogs with novel linkages |
US5292873A (en) | 1989-11-29 | 1994-03-08 | The Research Foundation Of State University Of New York | Nucleic acids labeled with naphthoquinone probe |
US5177198A (en) | 1989-11-30 | 1993-01-05 | University Of N.C. At Chapel Hill | Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates |
US5130302A (en) | 1989-12-20 | 1992-07-14 | Boron Bilogicals, Inc. | Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same |
US5486603A (en) | 1990-01-08 | 1996-01-23 | Gilead Sciences, Inc. | Oligonucleotide having enhanced binding affinity |
US5578718A (en) | 1990-01-11 | 1996-11-26 | Isis Pharmaceuticals, Inc. | Thiol-derivatized nucleosides |
US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
US6753423B1 (en) | 1990-01-11 | 2004-06-22 | Isis Pharmaceuticals, Inc. | Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals |
US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
US6395492B1 (en) | 1990-01-11 | 2002-05-28 | Isis Pharmaceuticals, Inc. | Derivatized oligonucleotides having improved uptake and other properties |
US5914396A (en) | 1990-01-11 | 1999-06-22 | Isis Pharmaceuticals, Inc. | 2'-O-modified nucleosides and phosphoramidites |
US6005087A (en) | 1995-06-06 | 1999-12-21 | Isis Pharmaceuticals, Inc. | 2'-modified oligonucleotides |
US5587361A (en) | 1991-10-15 | 1996-12-24 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
US5623065A (en) | 1990-08-13 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Gapped 2' modified oligonucleotides |
US5220007A (en) | 1990-02-15 | 1993-06-15 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of RNA and production of encoded polypeptides |
US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
WO1991013080A1 (en) | 1990-02-20 | 1991-09-05 | Gilead Sciences, Inc. | Pseudonucleosides and pseudonucleotides and their polymers |
US5214136A (en) | 1990-02-20 | 1993-05-25 | Gilead Sciences, Inc. | Anthraquinone-derivatives oligonucleotides |
US5321131A (en) | 1990-03-08 | 1994-06-14 | Hybridon, Inc. | Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling |
US5470967A (en) | 1990-04-10 | 1995-11-28 | The Dupont Merck Pharmaceutical Company | Oligonucleotide analogs with sulfamate linkages |
DK0455905T3 (da) | 1990-05-11 | 1998-12-07 | Microprobe Corp | Dipsticks til nukleinsyrehybridiseringsassays og fremgangsmåde til kovalent immobilisering af oligonukleotider |
WO1992000386A1 (en) | 1990-06-27 | 1992-01-09 | The Trustees Of Columbia University In The City Of New York | Methods for detecting spinal muscular atrophy type i and types ii/iii and marker therefor |
US5623070A (en) | 1990-07-27 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
US5608046A (en) | 1990-07-27 | 1997-03-04 | Isis Pharmaceuticals, Inc. | Conjugated 4'-desmethyl nucleoside analog compounds |
US5618704A (en) | 1990-07-27 | 1997-04-08 | Isis Pharmacueticals, Inc. | Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling |
US5218105A (en) | 1990-07-27 | 1993-06-08 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
US5688941A (en) | 1990-07-27 | 1997-11-18 | Isis Pharmaceuticals, Inc. | Methods of making conjugated 4' desmethyl nucleoside analog compounds |
US5677437A (en) | 1990-07-27 | 1997-10-14 | Isis Pharmaceuticals, Inc. | Heteroatomic oligonucleoside linkages |
US5541307A (en) | 1990-07-27 | 1996-07-30 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs and solid phase synthesis thereof |
US5138045A (en) | 1990-07-27 | 1992-08-11 | Isis Pharmaceuticals | Polyamine conjugated oligonucleotides |
JPH0874B2 (ja) | 1990-07-27 | 1996-01-10 | アイシス・ファーマシューティカルス・インコーポレーテッド | 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド |
US5489677A (en) | 1990-07-27 | 1996-02-06 | Isis Pharmaceuticals, Inc. | Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms |
WO1992002534A2 (en) | 1990-08-03 | 1992-02-20 | Sterling Drug, Inc. | Compounds and methods for inhibiting gene expression |
US5245022A (en) | 1990-08-03 | 1993-09-14 | Sterling Drug, Inc. | Exonuclease resistant terminally substituted oligonucleotides |
US5177196A (en) | 1990-08-16 | 1993-01-05 | Microprobe Corporation | Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof |
US5512667A (en) | 1990-08-28 | 1996-04-30 | Reed; Michael W. | Trifunctional intermediates for preparing 3'-tailed oligonucleotides |
US5214134A (en) | 1990-09-12 | 1993-05-25 | Sterling Winthrop Inc. | Process of linking nucleosides with a siloxane bridge |
US5561225A (en) | 1990-09-19 | 1996-10-01 | Southern Research Institute | Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages |
WO1992005186A1 (en) | 1990-09-20 | 1992-04-02 | Gilead Sciences | Modified internucleoside linkages |
US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
ATE198598T1 (de) | 1990-11-08 | 2001-01-15 | Hybridon Inc | Verbindung von mehrfachreportergruppen auf synthetischen oligonukleotiden |
JP3514779B2 (ja) | 1991-04-17 | 2004-03-31 | 株式会社アズウェル | 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 |
US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
WO1994008003A1 (en) | 1991-06-14 | 1994-04-14 | Isis Pharmaceuticals, Inc. | ANTISENSE OLIGONUCLEOTIDE INHIBITION OF THE ras GENE |
US5965722A (en) | 1991-05-21 | 1999-10-12 | Isis Pharmaceuticals, Inc. | Antisense inhibition of ras gene with chimeric and alternating oligonucleotides |
US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
US5371241A (en) | 1991-07-19 | 1994-12-06 | Pharmacia P-L Biochemicals Inc. | Fluorescein labelled phosphoramidites |
US5571799A (en) | 1991-08-12 | 1996-11-05 | Basco, Ltd. | (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response |
US5661134A (en) | 1991-10-15 | 1997-08-26 | Isis Pharmaceuticals, Inc. | Oligonucleotides for modulating Ha-ras or Ki-ras having phosphorothioate linkages of high chiral purity |
US6831166B2 (en) | 1992-10-23 | 2004-12-14 | Isis Pharmaceuticals, Inc. | Derivatized oligonucleotides having improved uptake and other properties |
US8153602B1 (en) | 1991-11-19 | 2012-04-10 | Isis Pharmaceuticals, Inc. | Composition and methods for the pulmonary delivery of nucleic acids |
US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
EP0618925B2 (en) | 1991-12-24 | 2012-04-18 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotides |
US5700922A (en) | 1991-12-24 | 1997-12-23 | Isis Pharmaceuticals, Inc. | PNA-DNA-PNA chimeric macromolecules |
US20060270624A1 (en) | 1991-12-24 | 2006-11-30 | Isis Pharmaceuticals, Inc. | Gapped 2' modified oligonucleotides |
US5595726A (en) | 1992-01-21 | 1997-01-21 | Pharmacyclics, Inc. | Chromophore probe for detection of nucleic acid |
US5565552A (en) | 1992-01-21 | 1996-10-15 | Pharmacyclics, Inc. | Method of expanded porphyrin-oligonucleotide conjugate synthesis |
US5633360A (en) | 1992-04-14 | 1997-05-27 | Gilead Sciences, Inc. | Oligonucleotide analogs capable of passive cell membrane permeation |
US5434257A (en) | 1992-06-01 | 1995-07-18 | Gilead Sciences, Inc. | Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages |
US5272250A (en) | 1992-07-10 | 1993-12-21 | Spielvogel Bernard F | Boronated phosphoramidate compounds |
US5652355A (en) | 1992-07-23 | 1997-07-29 | Worcester Foundation For Experimental Biology | Hybrid oligonucleotide phosphorothioates |
TW244371B (da) | 1992-07-23 | 1995-04-01 | Tri Clover Inc | |
US6346614B1 (en) | 1992-07-23 | 2002-02-12 | Hybridon, Inc. | Hybrid oligonucleotide phosphorothioates |
RU95104940A (ru) | 1992-07-27 | 1997-01-10 | Хайбрайдон | Способ введения в олигонуклеотид алкилфосфонотиоатной или арилфосфонотиоатной межнуклеотидной связи, способ получения олигонуклеотида, олигонуклеотиды, способ ингибирования генной экспрессии, способ лечения |
US5574142A (en) | 1992-12-15 | 1996-11-12 | Microprobe Corporation | Peptide linkers for improved oligonucleotide delivery |
DE69400208T2 (de) | 1993-01-25 | 1996-11-28 | Hybridon Inc | Olionukleotidalkylphosphonate und -phosphonothioate |
US5476925A (en) | 1993-02-01 | 1995-12-19 | Northwestern University | Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups |
GB9304618D0 (en) | 1993-03-06 | 1993-04-21 | Ciba Geigy Ag | Chemical compounds |
EP0691977B1 (en) | 1993-03-31 | 1997-11-26 | Sanofi | Oligonucleotides with amide linkages replacing phosphodiester linkages |
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 |
US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
DE69425903T2 (de) | 1993-12-09 | 2001-02-15 | Thomas Jefferson University Ph | Verbindungen und verfahren zur ortsspezifischen mutation in eukaryotischen zellen |
US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
US5625050A (en) | 1994-03-31 | 1997-04-29 | Amgen Inc. | Modified oligonucleotides and intermediates useful in nucleic acid therapeutics |
US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
FR2720757B1 (fr) | 1994-06-03 | 1996-08-02 | Inst Nat Sante Rech Med | Procédé et sondes pour la détection de marqueurs liés au locus des amyotrophies spinales infantiles. |
US5597696A (en) | 1994-07-18 | 1997-01-28 | Becton Dickinson And Company | Covalent cyanine dye oligonucleotide conjugates |
US5580731A (en) | 1994-08-25 | 1996-12-03 | Chiron Corporation | N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith |
EP0708178A1 (en) | 1994-10-19 | 1996-04-24 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Survival motor neuron (SMN) gene: a gene for spinal muscular atrophy |
US6608035B1 (en) | 1994-10-25 | 2003-08-19 | Hybridon, Inc. | Method of down-regulating gene expression |
US5652356A (en) | 1995-08-17 | 1997-07-29 | Hybridon, Inc. | Inverted chimeric and hybrid oligonucleotides |
US8217015B2 (en) | 2003-04-04 | 2012-07-10 | Arrowhead Madison Inc. | Endosomolytic polymers |
JP4293636B2 (ja) | 1996-02-14 | 2009-07-08 | アイシス・ファーマシューティカルス・インコーポレーテッド | 糖修飾ギャップ付オリゴヌクレオチド |
US6015710A (en) | 1996-04-09 | 2000-01-18 | The University Of Texas System | Modulation of mammalian telomerase by peptide nucleic acids |
US5898031A (en) | 1996-06-06 | 1999-04-27 | Isis Pharmaceuticals, Inc. | Oligoribonucleotides for cleaving RNA |
US5912332A (en) | 1996-07-26 | 1999-06-15 | Hybridon, Inc. | Affinity-based purification of oligonucleotides using soluble multimeric oligonucleotides |
AU8285998A (en) | 1997-07-02 | 1999-01-25 | Sdg, Inc. | Targeted liposomal constructs for diagnostic and therapeutic uses |
JPH1133863A (ja) | 1997-07-23 | 1999-02-09 | Howa Mach Ltd | 工具交換装置及び工具交換用の押出し装置 |
US6794499B2 (en) * | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
US7572582B2 (en) | 1997-09-12 | 2009-08-11 | Exiqon A/S | Oligonucleotide analogues |
US7715989B2 (en) | 1998-04-03 | 2010-05-11 | Elitech Holding B.V. | Systems and methods for predicting oligonucleotide melting temperature (TmS) |
EP1088066B1 (en) | 1998-06-19 | 2006-11-29 | McGILL UNIVERSITY | Antisense oligonucleotide constructs based on beta-arabinofuranose and its analogues |
US6503754B1 (en) | 2000-09-07 | 2003-01-07 | Isis Pharmaceuticals, Inc. | Antisense modulation of BH3 interacting domain death agonist expression |
US6646113B1 (en) * | 1998-09-17 | 2003-11-11 | The Trustees Of The University Of Pennsylvania | Nucleic acid molecule encoding human survival of motor neuron-interacting protein 1 (SIP1) deletion mutants |
US6210892B1 (en) | 1998-10-07 | 2001-04-03 | Isis Pharmaceuticals, Inc. | Alteration of cellular behavior by antisense modulation of mRNA processing |
EP0999270A1 (en) | 1998-10-09 | 2000-05-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Method of regulating SMN gene expression |
US6465628B1 (en) | 1999-02-04 | 2002-10-15 | Isis Pharmaceuticals, Inc. | Process for the synthesis of oligomeric compounds |
BRPI0008131B8 (pt) | 1999-02-12 | 2021-05-25 | Daiichi Sankyo Co Ltd | composto ou um sal deste, análogo de oligonucleotídeo, composição farmacêutica, sonda para um gene,iniciador para começar a amplificação, uso de um análogo de oligonucleotídeo ou de um sal deste farmacologicamente aceitável, agente antisentido, e, agente antígeno |
US9029523B2 (en) | 2000-04-26 | 2015-05-12 | Ceres, Inc. | Promoter, promoter control elements, and combinations, and uses thereof |
US20080281041A1 (en) | 1999-06-07 | 2008-11-13 | Rozema David B | Reversibly Masked Polymers |
US20040002153A1 (en) | 1999-07-21 | 2004-01-01 | Monia Brett P. | Modulation of PTEN expression via oligomeric compounds |
US6284538B1 (en) | 1999-07-21 | 2001-09-04 | Isis Pharmaceuticals, Inc. | Antisense inhibition of PTEN expression |
AU6492400A (en) | 1999-07-22 | 2001-02-13 | Genaissance Pharmaceuticals, Inc. | Drug target isogenes: polymorphisms in the prostaglandin-endoperoxide synthase 2gene |
US6677445B1 (en) | 1999-08-27 | 2004-01-13 | Chiron Corporation | Chimeric antisense oligonucleotides and cell transfecting formulations thereof |
IL132972A0 (en) | 1999-11-16 | 2001-03-19 | Yissum Res Dev Co | Pharmaceutical compositions comprising acetylcholinesterase antisense deoxynucleotides for the treatment of muscular and neuromuscular disorders |
US6187545B1 (en) * | 2000-01-19 | 2001-02-13 | Isis Pharmaceuticals Inc. | Antisense modulation of pepck-cytosolic expression |
US6287860B1 (en) | 2000-01-20 | 2001-09-11 | Isis Pharmaceuticals, Inc. | Antisense inhibition of MEKK2 expression |
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 |
NZ521698A (en) | 2000-03-27 | 2004-08-27 | Univ Delaware | Targeted chromosomal genomic alterations with modified single stranded oligonucleotides |
JP2004509604A (ja) | 2000-03-28 | 2004-04-02 | アイシス・ファーマシューティカルス・インコーポレーテッド | mRNAプロセッシングのアンチセンスモジュレーションによる、細胞行動の改変 |
US20040241651A1 (en) | 2000-04-07 | 2004-12-02 | Alexander Olek | Detection of single nucleotide polymorphisms (snp's) and cytosine-methylations |
KR20020097241A (ko) | 2000-05-04 | 2002-12-31 | 에이브이아이 바이오파마 인코포레이티드 | 스플라이스-영역 안티센스 조성물 및 방법 |
WO2001091699A2 (en) | 2000-05-30 | 2001-12-06 | Advanced Research & Technology Institute | Compositions and methods for identifying agents which modulate pten function and pi-3 kinase pathways |
US6727355B2 (en) | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
US20050054836A1 (en) | 2000-11-09 | 2005-03-10 | Cold Spring Harbor Laboratory | Chimeric molecules to modulate gene expression |
EP1345959B1 (en) | 2000-11-20 | 2011-05-25 | The Board Of Trustees Of The University Of Illinois | Membrane scaffold proteins |
US20040058356A1 (en) | 2001-03-01 | 2004-03-25 | Warren Mary E. | Methods for global profiling gene regulatory element activity |
US6825338B2 (en) * | 2001-03-30 | 2004-11-30 | Isis Pharmaceuticals, Inc. | Labeled oligonucleotides, methods for making same, and compounds useful therefor |
CA2446112C (en) | 2001-05-08 | 2011-04-26 | Darwin Molecular Corporation | A method for regulating immune function in primates using the foxp3 protein |
US20050176025A1 (en) | 2001-05-18 | 2005-08-11 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of B-cell CLL/Lymphoma-2 (BCL-2) gene expression using short interfering nucleic acid (siNA) |
AU2002344853A1 (en) | 2001-06-14 | 2003-01-02 | Active Pass Pharmaceuticals, Inc. | Abca10 transporter |
US7407943B2 (en) | 2001-08-01 | 2008-08-05 | Isis Pharmaceuticals, Inc. | Antisense modulation of apolipoprotein B expression |
US7259150B2 (en) | 2001-08-07 | 2007-08-21 | Isis Pharmaceuticals, Inc. | Modulation of apolipoprotein (a) expression |
WO2003037909A1 (en) | 2001-10-29 | 2003-05-08 | Mcgill University | Acyclic linker-containing oligonucleotides and uses thereof |
US20030219770A1 (en) * | 2001-11-08 | 2003-11-27 | Eshleman James R. | Methods and systems of nucleic acid sequencing |
US20030198983A1 (en) | 2002-02-01 | 2003-10-23 | Affymetrix, Inc. | Methods of genetic analysis of human genes |
US20080207542A1 (en) | 2002-03-26 | 2008-08-28 | Sirna Therapeutics, Inc. | RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA) |
AU2003225495B2 (en) | 2002-04-05 | 2009-01-15 | Roche Innovation Center Copenhagen A/S | Oligomeric compounds for the modulation HIF-1alpha expression |
US20050130924A1 (en) | 2002-06-26 | 2005-06-16 | Monia Brett P. | Antisense inhibition via RNAse H-independent reduction in mRNA |
WO2004011613A2 (en) | 2002-07-29 | 2004-02-05 | Epigenesis Pharmaceuticals, Inc. | Composition & methods for treatment and screening |
US20050003369A1 (en) | 2002-10-10 | 2005-01-06 | Affymetrix, Inc. | Method for depleting specific nucleic acids from a mixture |
US20040219565A1 (en) | 2002-10-21 | 2004-11-04 | Sakari Kauppinen | Oligonucleotides useful for detecting and analyzing nucleic acids of interest |
AU2003290596B2 (en) * | 2002-11-05 | 2011-05-12 | Isis Pharmaceuticals, Inc. | 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 |
JP2006507841A (ja) | 2002-11-14 | 2006-03-09 | ダーマコン, インコーポレイテッド | 機能的siRNAおよび超機能的siRNA |
US7655785B1 (en) | 2002-11-14 | 2010-02-02 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof |
DK1569661T3 (da) | 2002-11-18 | 2010-01-11 | Santaris Pharma As | Antisense design |
CA2515644A1 (en) | 2003-02-10 | 2004-08-19 | Santaris Pharma A/S | Oligomeric compounds for the modulation of ras expression |
ES2576677T3 (es) | 2003-03-21 | 2016-07-08 | Roche Innovation Center Copenhagen A/S | Análogos de ARN interfirientes cortos |
FR2853663B1 (fr) | 2003-04-14 | 2007-08-31 | Aventis Pharma Sa | Procede d'obtention de lignees de mastocytes a partir de tissus de porcs et procede de production de molecules de type heparine |
US8092992B2 (en) | 2003-05-29 | 2012-01-10 | Salk Institute For Biological Studies | Transcriptional regulation of gene expression by small double-stranded modulatory RNA |
WO2004113867A2 (en) | 2003-06-16 | 2004-12-29 | University Of Massachusetts | Exon analysis |
EP2530157B1 (en) | 2003-07-31 | 2016-09-28 | Regulus Therapeutics Inc. | Oligomeric compounds and compositions for use in modulation of miRNAs |
US7888497B2 (en) | 2003-08-13 | 2011-02-15 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof |
US20050108783A1 (en) * | 2003-09-23 | 2005-05-19 | Chihiro Koike | Porcine invariant chain protein, full length cDNA, genomic organization, and regulatory region |
WO2005044981A2 (en) | 2003-10-23 | 2005-05-19 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of gene expression using short interfering nucleic acid (sina) |
GB0324854D0 (en) | 2003-10-24 | 2003-11-26 | Expresson Biosystems Ltd | App/ena antisense |
UA88457C2 (ru) | 2003-10-30 | 2009-10-26 | Коли Фармасьютикал Гмбх | Иммуностимулирующая нуклеиновая кислота с улучшенной иммуностимулирующей эффективностью |
EP1756137A4 (en) | 2003-11-05 | 2007-10-31 | Univ Texas | DIAGNOSTIC AND THERAPEUTIC PROCEDURES AND COMPOSITIONS CONCERNING PTEN AND BREAST CANCER |
EP1706489B9 (en) | 2003-12-23 | 2011-01-05 | Santaris Pharma A/S | Oligomeric compounds for the modulation of bcl-2 |
US20050244851A1 (en) | 2004-01-13 | 2005-11-03 | Affymetrix, Inc. | Methods of analysis of alternative splicing in human |
WO2005072527A2 (en) | 2004-01-23 | 2005-08-11 | Avi Biopharma, Inc. | Antisense oligomers and methods for inducing immune tolerance and immunosuppression |
US20050164209A1 (en) | 2004-01-23 | 2005-07-28 | Bennett C. F. | Hepatocyte free uptake assays |
EP1713912B1 (en) | 2004-01-30 | 2013-09-18 | Santaris Pharma A/S | Modified short interfering rna (modified sirna) |
WO2005089169A2 (en) | 2004-03-12 | 2005-09-29 | Exelixis, Inc. | Mptens as modifiers of the pten pathway and methods of use |
US8314226B2 (en) | 2004-03-29 | 2012-11-20 | The General Hospital Corporation | Oligonucleotide complex compositions and methods of use as gene alteration tools |
US7374927B2 (en) | 2004-05-03 | 2008-05-20 | Affymetrix, Inc. | Methods of analysis of degraded nucleic acid samples |
CN101052717A (zh) | 2004-05-11 | 2007-10-10 | α基因株式会社 | 诱发rna干扰的多核苷酸以及使用该多核苷酸的基因表达抑制方法 |
US7687616B1 (en) | 2004-05-14 | 2010-03-30 | Rosetta Genomics Ltd | Small molecules modulating activity of micro RNA oligonucleotides and micro RNA targets and uses thereof |
US20050265927A1 (en) | 2004-05-17 | 2005-12-01 | Yale University | Intranasal delivery of nucleic acid molecules |
ES2361325T3 (es) | 2004-06-28 | 2011-06-16 | The University Of Western Australia | Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos. |
US8029985B2 (en) | 2004-09-01 | 2011-10-04 | Vybion, Inc. | Amplified bioassay |
US7838657B2 (en) * | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
WO2006063356A1 (en) | 2004-12-10 | 2006-06-15 | Isis Phamaceuticals, Inc. | Regulation of epigenetic control of gene expression |
US7879992B2 (en) | 2005-01-31 | 2011-02-01 | Isis Pharmaceuticals, Inc. | Modification of MyD88 splicing using modified oligonucleotides |
AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
WO2006130201A1 (en) | 2005-03-14 | 2006-12-07 | Board Of Regents, The University Of Texas System | Antigene oligomers inhibit transcription |
US7449297B2 (en) * | 2005-04-14 | 2008-11-11 | Euclid Diagnostics Llc | Methods of copying the methylation pattern of DNA during isothermal amplification and microarrays |
JP5362350B2 (ja) | 2005-04-15 | 2013-12-11 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 小分子活性化rna分子及び使用方法 |
US8586719B2 (en) | 2005-04-27 | 2013-11-19 | Pacific Arrow Limited | Triterpenes for modulating gene expression and cell membrane, and as antiprotozoal agents |
ES2545223T3 (es) * | 2005-06-23 | 2015-09-09 | Isis Pharmaceuticals, Inc. | Composiciones y procedimientos para modular el corte y empalme de SMN2 |
US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
DK2641970T3 (da) | 2005-11-17 | 2015-02-02 | Univ Texas | Modulation af genekspression med oligomerer targeteret til kromosomalt DNA |
JP5078904B2 (ja) | 2005-11-21 | 2012-11-21 | ジヨンソン・アンド・ジヨンソン・リサーチ・ピーテイワイ・リミテツド | 多標的干渉rnaならびにそれらの使用および設計方法 |
CN103301475B (zh) | 2005-12-28 | 2016-08-03 | 斯克里普斯研究所 | 药物组合物和表达载体以及调节基因表达的方法和核酸分子的应用 |
WO2007133812A2 (en) | 2005-12-30 | 2007-11-22 | Philadelphia Health & Education Corporation, D/B/A Drexel University College Of Medicine | Improved carriers for delivery of nucleic acid agents to cells and tissues |
US7569686B1 (en) | 2006-01-27 | 2009-08-04 | Isis Pharmaceuticals, Inc. | Compounds and methods for synthesis of bicyclic nucleic acid analogs |
DK1984381T3 (da) | 2006-01-27 | 2010-11-01 | Isis Pharmaceuticals Inc | 6-modificerede bicycliske nukleinsyreanaloger |
EP2388328A1 (en) | 2006-01-27 | 2011-11-23 | Isis Pharmaceuticals, Inc. | Oligomeric compounds and compositions for the use in modulation of micrornas |
EP1989307B1 (en) | 2006-02-08 | 2012-08-08 | Quark Pharmaceuticals, Inc. | NOVEL TANDEM siRNAS |
CA2649045C (en) | 2006-04-03 | 2019-06-11 | Santaris Pharma A/S | Pharmaceutical composition comprising anti-mirna antisense oligonucleotides |
US8226930B2 (en) | 2006-04-07 | 2012-07-24 | Franco Antonio Laccone | Synthetic MeCP2 sequence for protein substitution therapy |
WO2007143315A2 (en) | 2006-05-05 | 2007-12-13 | Isis Pharmaceutical, Inc. | Compounds and methods for modulating expression of pcsk9 |
WO2008002996A2 (en) | 2006-06-27 | 2008-01-03 | Shanghai Institutes For Biological Sciences | Rheumatoid arthritis t cell vaccine |
KR101133799B1 (ko) | 2006-08-18 | 2012-04-24 | 에프. 호프만-라 로슈 아게 | 폴리뉴클레오타이드의 생체내 전달을 위한 다가접합체 |
EP2054529B1 (en) | 2006-08-25 | 2014-03-26 | Duke University | Methods for in vivo identification of endogenous mrna targets of micrornas |
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 |
WO2008029619A1 (fr) | 2006-09-07 | 2008-03-13 | Daiichi Sankyo Company, Limited | Oligonucléotide antisens ena ayant une action spécifique de la séquence |
US20080176793A1 (en) | 2006-09-18 | 2008-07-24 | The Regents Of The University Of California | Upregulating activity or expression of bdnf to mitigate cognitive impairment in asymptomatic huntington's subjects |
JP5665317B2 (ja) | 2006-10-18 | 2015-02-04 | アイシス ファーマシューティカルズ, インコーポレーテッド | アンチセンス化合物 |
JP2010509923A (ja) | 2006-11-23 | 2010-04-02 | ミルクス セラピューティクス アンパーツゼルスカブ | 標的rnaの活性を変化させるためのオリゴヌクレオチド |
EP2064350B1 (en) | 2006-11-27 | 2013-01-02 | Ludwig Institute for Cancer Research Ltd. | Expression of foxp3 by cancer cells |
US8058003B2 (en) | 2007-01-19 | 2011-11-15 | The Regents Of The University Of Michigan | ADRB2 cancer markers |
ITMI20070127A1 (it) | 2007-01-29 | 2008-07-30 | Fond I R C C S | Proteine e-o peptidi per la prevenzione e-o cura di malattie neurodegenerative |
WO2008103761A2 (en) | 2007-02-20 | 2008-08-28 | Sequenom, Inc. | Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation |
US7858592B2 (en) | 2007-02-26 | 2010-12-28 | The Board Of Regents Of The University Of Texas System | Interfering RNAs against the promoter region of P53 |
WO2008113832A2 (en) | 2007-03-22 | 2008-09-25 | Santaris Pharma A/S | SHORT RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF TARGET mRNA |
US8158596B2 (en) | 2007-05-11 | 2012-04-17 | The Regents Of The University Of Michigan | Materials and methods for FOXP3 tumor suppression |
EP2714904B1 (en) | 2007-06-14 | 2017-04-12 | Mirx Therapeutics ApS | Oligonucleotides for modulation of target rna activity |
US20090082297A1 (en) | 2007-06-25 | 2009-03-26 | Lioy Daniel T | Compositions and Methods for Regulating Gene Expression |
ES2694726T3 (es) * | 2007-06-29 | 2018-12-26 | Sarepta Therapeutics, Inc. | Conjugados peptídicos específicos de tejido y métodos |
EP2195029A2 (en) | 2007-08-24 | 2010-06-16 | Oryzon Genomics SA | Treatment and prevention of neurodegenerative diseases |
EP3492594A1 (en) | 2007-10-04 | 2019-06-05 | Roche Innovation Center Copenhagen A/S | Micromirs |
JP2010539990A (ja) | 2007-10-04 | 2010-12-24 | ボード オブ リージェンツ ザ ユニバーシティー オブ テキサス システム | アンチセンス転写物を標的とするagRNAおよびギャップマーを用いた遺伝子発現の調節方法 |
ITRM20070523A1 (it) | 2007-10-05 | 2009-04-06 | Consiglio Nazionale Ricerche | Acido nucleico codificante per una proteina regolatrice specifica della trascrizione dell'utrofina proteina da esso codificata e sue applicazioni |
US9029337B2 (en) | 2007-11-09 | 2015-05-12 | Isis Pharmaceuticals, Inc. | Modulation of factor 7 expression |
EP2219680A2 (en) | 2007-11-13 | 2010-08-25 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating protein expression |
US8937217B2 (en) | 2007-12-18 | 2015-01-20 | E. I. Du Pont De Nemours And Company | Down-regulation of gene expression using artificial microRNAs |
WO2009090182A1 (en) | 2008-01-14 | 2009-07-23 | Santaris Pharma A/S | C4'-substituted - dna nucleotide gapmer oligonucleotides |
AU2009221064B2 (en) | 2008-03-07 | 2014-12-11 | Roche Innovation Center Copenhagen A/S | Pharmaceutical compositions for treatment of microRNA related diseases |
AU2009223615B2 (en) | 2008-03-07 | 2014-09-11 | Senesco Technologies, Inc. | Use of siRNA to achieve down regulation of an endogenous gene in combination with the use of a sense construct to achieve expression of a desired polynucleotide |
US8846639B2 (en) | 2008-04-04 | 2014-09-30 | Isis Pharmaceutical, Inc. | Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity |
AU2009235941A1 (en) | 2008-04-07 | 2009-10-15 | Riken | RNA molecules and uses thereof |
US8916532B2 (en) | 2008-04-28 | 2014-12-23 | The Trustees Of The University Of Pennsylvania | Methods for enhancing utrophin production via inhibition of microRNA |
US8633019B2 (en) | 2008-05-27 | 2014-01-21 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
WO2009149182A1 (en) | 2008-06-04 | 2009-12-10 | The Board Of Regents Of The University Of Texas System | Modulation of gene expression through endogenous small rna targeting of gene promoters |
CA2635187A1 (en) | 2008-06-05 | 2009-12-05 | The Royal Institution For The Advancement Of Learning/Mcgill University | Oligonucleotide duplexes and uses thereof |
EP2310505B1 (en) | 2008-06-30 | 2017-08-09 | Roche Innovation Center Copenhagen A/S | Antidote oligomers |
JP2011529686A (ja) | 2008-07-29 | 2011-12-15 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | ポリグルタミンタンパク質発現の選択的阻害 |
JP2012503651A (ja) | 2008-09-26 | 2012-02-09 | エージェンシー フォー サイエンス,テクノロジー アンド リサーチ | 3−デアザネプラノシン誘導体 |
JP6128732B2 (ja) | 2008-10-03 | 2017-05-17 | クルナ・インコーポレーテッド | アポリポタンパク質−a1に対する天然アンチセンス転写物の抑制によるアポリポタンパク質−a1関連疾患の治療 |
KR20110071017A (ko) | 2008-10-16 | 2011-06-27 | 마리나 바이오테크, 인크. | 유전자 침묵 치료제의 리포좀에 의한 효율적인 전달을 위한 프로세스 및 조성물 |
EP2358876A1 (en) | 2008-11-17 | 2011-08-24 | F. Hoffmann-La Roche AG | Compositions and methods for inhibiting expression of factor vii genes |
GB0821457D0 (en) | 2008-11-24 | 2008-12-31 | Trillion Genomics Ltd | Oligonucleotides |
US20110237649A1 (en) | 2008-12-04 | 2011-09-29 | Opko Curna, Llc | Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirtuin 1 |
KR101761424B1 (ko) | 2008-12-04 | 2017-07-26 | 큐알엔에이, 인크. | Vegf에 대한 천연 안티센스 전사체의 억제에 의해 맥관 내피 성장 인자(vegf) 관련된 질환의 치료 |
RU2620970C2 (ru) | 2008-12-04 | 2017-05-30 | КьюРНА,Инк., | Лечение связанных с эритропоэтином (еро) заболеваний путем ингибирования природного антисмыслового транскрипта к еро |
CN102361985B (zh) | 2008-12-04 | 2017-06-20 | 库尔纳公司 | 通过抑制肿瘤抑制基因的天然反义转录物治疗肿瘤抑制基因相关性疾病 |
EP2405921A4 (en) | 2009-01-26 | 2013-05-22 | Protiva Biotherapeutics Inc | COMPOSITIONS AND METHODS FOR INACTIVATION OF APOLIPOPROTEIN C-III EXPRESSION |
ES2560107T3 (es) | 2009-02-12 | 2016-02-17 | Curna, Inc. | Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado de cerebro (BDNF) por inhibición de transcrito antisentido natural para BDNF |
ES2658626T3 (es) | 2009-02-12 | 2018-03-12 | Curna, Inc. | Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado de células gliales (GDNF) por inhibición de transcrito antisentido natural a GDNF |
US20120040857A1 (en) | 2009-02-13 | 2012-02-16 | The General Hospital Corporation | Isolation of factors that associate directly or indirectly with chromatin |
EP2963116B1 (en) | 2009-03-04 | 2020-11-11 | CuRNA, Inc. | Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirt 1 |
CA2755409C (en) | 2009-03-16 | 2019-04-30 | Joseph Collard | Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2 |
CA2755404C (en) | 2009-03-17 | 2020-03-24 | Joseph Collard | Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1 |
CA2758189C (en) | 2009-04-10 | 2020-12-29 | Association Institut De Myologie | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
WO2010124231A2 (en) | 2009-04-24 | 2010-10-28 | The Board Of Regents Of The University Of Texas System | Modulation of gene expression using oligomers that target gene regions downstream of 3' untranslated regions |
NO2424987T3 (da) | 2009-05-01 | 2018-04-14 | ||
CN106237345A (zh) | 2009-05-06 | 2016-12-21 | 库尔纳公司 | 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病 |
US20120046236A1 (en) | 2009-05-06 | 2012-02-23 | Opko Curna Llc | Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp |
CA3185821A1 (en) | 2009-05-08 | 2010-11-11 | Curna, Inc. | Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family |
CA2762369C (en) | 2009-05-18 | 2021-12-28 | Joseph Collard | Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor |
WO2010135695A2 (en) | 2009-05-22 | 2010-11-25 | Curna, Inc. | TREATMENT OF TRANSCRIPTION FACTOR E3 (TFE3) and INSULIN RECEPTOR SUBSTRATE 2 (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO TFE3 |
ES2618576T3 (es) | 2009-05-28 | 2017-06-21 | Curna, Inc. | Tratamiento de enfermedades relacionadas con un gen antivírico mediante la inhibición de una transcripción antisentido natural a un gen antivírico |
CN102612560B (zh) | 2009-06-16 | 2017-10-17 | 库尔纳公司 | 通过抑制针对对氧磷酶1(pon1)的天然反义转录物来治疗pon1相关的疾病 |
EP2443237B1 (en) | 2009-06-16 | 2017-02-22 | CuRNA, Inc. | Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene |
US8859515B2 (en) | 2009-06-24 | 2014-10-14 | Curna, Inc. | Treatment of tumor necrosis factor receptor 2 (TNFR2) related diseases by inhibition of natural antisense transcript to TNFR2 |
CN102482672B (zh) | 2009-06-26 | 2016-11-09 | 库尔纳公司 | 通过抑制唐氏综合征基因的天然反义转录物治疗唐氏综合征基因相关疾病 |
WO2011005786A2 (en) | 2009-07-06 | 2011-01-13 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for enhancing production of a biological product |
CA2768598A1 (en) | 2009-07-22 | 2011-01-27 | Cenix Bioscience Gmbh | Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes |
EP2458005A1 (en) | 2009-07-23 | 2012-05-30 | Galaxy Pharma Inc. | Fgf21 cis-element binding substance |
KR101801407B1 (ko) | 2009-07-24 | 2017-11-24 | 큐알엔에이, 인크. | 시르투인 (sirt)에 대한 천연 안티센스 전사체의 억제에 의한 시르투인 관련된 질환의 치료 |
CA2769665A1 (en) | 2009-08-05 | 2011-02-10 | Opko Curna, Llc | Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins) |
US9044493B2 (en) | 2009-08-11 | 2015-06-02 | Curna, Inc. | Treatment of Adiponectin related diseases by inhibition of natural antisense transcript to an Adiponectin |
KR101805213B1 (ko) | 2009-08-21 | 2017-12-06 | 큐알엔에이, 인크. | Chip에 대한 천연 안티센스 전사체의 억제에 의한 “hsp70-상호작용 단백질(chip)의 c-말단” 관련된 질환의 치료 |
JP5964232B2 (ja) | 2009-08-25 | 2016-08-03 | カッパーアールエヌエー,インコーポレイテッド | ‘iqモチーフ含有gtpアーゼ活性化タンパク質’(iqgap)に対する天然アンチセンス転写産物の阻害によるiqgap関連疾患の治療 |
WO2011025862A2 (en) | 2009-08-28 | 2011-03-03 | Curna, Inc. | Treatment of atp-binding cassette sub-family b member 1 (abcb1) related diseases by inhibition of natural antisense transcript to abcb1 |
WO2011032109A1 (en) * | 2009-09-11 | 2011-03-17 | Sma Foundation | Biomarkers for spinal muscular atrophy |
WO2011038205A2 (en) | 2009-09-25 | 2011-03-31 | Curna, Inc. | Treatment of growth hormone (gh) related diseases by inhibition of natural antisense transcript to gh |
CA2775111C (en) | 2009-09-25 | 2019-12-31 | Opko Curna, Llc | Treatment of filaggrin (flg) related diseases by modulation of flg expression and activity |
EP2490699A1 (en) | 2009-10-20 | 2012-08-29 | Santaris Pharma A/S | Oral delivery of therapeutically effective lna oligonucleotides |
KR101138048B1 (ko) | 2009-11-06 | 2012-04-23 | 성균관대학교산학협력단 | Bdnf의 발현을 증가시키는 신규 펩타이드 및 이를 포함하는 알츠하이머병 또는 파킨슨병의 예방 및 치료용 약학 조성물 |
KR101823702B1 (ko) | 2009-12-16 | 2018-01-30 | 큐알엔에이, 인크. | 막 결합 전사 인자 펩티다제, 부위 1(mbtps1)에 대한 천연 안티센스 전사체의 억제에 의한 mbtps1 관련 질환의 치료 |
RU2609631C2 (ru) | 2009-12-23 | 2017-02-02 | Курна, Инк. | Лечение заболеваний, связанных с фактором роста гепатоцитов (фрг), посредством ингибирования природного антисмыслового транскрипта к фрг |
KR101793753B1 (ko) | 2009-12-23 | 2017-11-03 | 큐알엔에이, 인크. | 커플링방지 단백질 2(ucp2)에 대한 천연 안티센스 전사체의 저해에 의한 ucp2 관련 질환의 치료 |
US8921334B2 (en) | 2009-12-29 | 2014-12-30 | Curna, Inc. | Treatment of nuclear respiratory factor 1 (NRF1) related diseases by inhibition of natural antisense transcript to NRF1 |
RU2611186C2 (ru) | 2009-12-29 | 2017-02-21 | Курна, Инк. | ЛЕЧЕНИЕ ЗАБОЛЕВАНИЙ, СВЯЗАННЫХ С ОПУХОЛЕВЫМ БЕЛКОМ 63 (р63), ПУТЕМ ИНГИБИРОВАНИЯ ПРИРОДНОГО АНТИСМЫСЛОВОГО ТРАНСКРИПТА К р63 |
JP6083735B2 (ja) | 2009-12-31 | 2017-02-22 | カッパーアールエヌエー,インコーポレイテッド | インスリン受容体基質2(irs2)および転写因子3(tfe3)に対する天然アンチセンス転写物の阻害によるインスリン受容体基質2(irs2)関連疾患の治療 |
JP5886757B2 (ja) | 2010-01-04 | 2016-03-16 | カッパーアールエヌエー,インコーポレイテッド | インターフェロン調節因子8(irf8)に対する天然アンチセンス転写物の阻害によるインターフェロン調節因子8(irf8)関連疾患の治療 |
RU2612161C2 (ru) | 2010-01-06 | 2017-03-02 | Курна, Инк. | Лечение заболеваний, связанных с геном развития поджелудочной железы, путем ингибирования природного антисмыслового транскрипта к гену развития поджелудочной железы |
KR101854926B1 (ko) | 2010-01-11 | 2018-05-04 | 큐알엔에이, 인크. | 성 호르몬 결합 글로불린 (shbg)에 대한 자연 안티센스 전사체의 저해에 의한 성 호르몬 결합 글로불린 (shbg) 관련된 질환의 치료 |
CA2786568A1 (en) | 2010-01-25 | 2011-07-28 | Curna, Inc. | Treatment of rnase h1 related diseases by inhibition of natural antisense transcript to rnase h1 |
WO2011097388A1 (en) | 2010-02-03 | 2011-08-11 | Alnylam Pharmaceuticals, Inc. | Selective inhibition of polyglutamine protein expression |
WO2011097582A2 (en) | 2010-02-08 | 2011-08-11 | Opko Curna Llc | Treatment of arachidonate 12-lipoxygenase, 12r type (alox12b) related diseases by inhibition of natural antisense transcript to alox12b |
WO2011097641A1 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
CA2790506A1 (en) | 2010-02-22 | 2011-08-25 | Curna, Inc. | Treatment of pyrroline-5-carboxylate reductase 1 (pycr1) related diseases by inhibition of natural antisense transcript to pycr1 |
US20130079505A1 (en) | 2010-03-24 | 2013-03-28 | Mirrx Therapeutics A/S | Bivalent antisense oligonucleotides |
CA2795145C (en) | 2010-04-02 | 2019-01-22 | Curna, Inc. | Treatment of colony-stimulating factor 3 (csf3) related diseases by inhibition of natural antisense transcript to csf3 |
WO2011127337A2 (en) | 2010-04-09 | 2011-10-13 | Opko Curna Llc | Treatment of fibroblast growth factor 21 (fgf21) related diseases by inhibition of natural antisense transcript to fgf21 |
US9145556B2 (en) | 2010-04-13 | 2015-09-29 | Life Technologies Corporation | Compositions and methods for inhibition of nucleic acids function |
JP2013525483A (ja) | 2010-05-03 | 2013-06-20 | カッパーアールエヌエー,インコーポレイテッド | サーチュイン(sirt)に対する天然アンチセンス転写物の阻害によるサーチュイン(sirt)関連疾患の治療 |
TWI586356B (zh) | 2010-05-14 | 2017-06-11 | 可娜公司 | 藉由抑制par4天然反股轉錄本治療par4相關疾病 |
WO2011146675A2 (en) | 2010-05-19 | 2011-11-24 | Opko Curna Llc | Treatment of lim homeobox 2 (lhx2) related diseases by inhibition of natural antisense transcript to lhx2 |
TW201201819A (en) | 2010-05-19 | 2012-01-16 | Opko Curna Llc | Treatment of BCL2-like 11 (BCL2L11) related diseases by inhibition of natural antisense transcript to BCL2L11 |
EP2576783B1 (en) | 2010-05-26 | 2017-11-29 | CuRNA, Inc. | Treatment of atonal homolog 1 (atoh1) related diseases by inhibition of natural antisense transcript to atoh1 |
RU2620978C2 (ru) | 2010-05-26 | 2017-05-30 | Курна, Инк. | Лечение заболеваний, связанных с метионинсульфоксидредуктазой а (msra), путем ингибирования природного антисмыслового транскрипта гена msra |
WO2011159836A2 (en) | 2010-06-15 | 2011-12-22 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating interaction between proteins and target nucleic acids |
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 |
GB201010557D0 (en) | 2010-06-23 | 2010-08-11 | Mina Therapeutics Ltd | RNA molecules and uses thereof |
EP2585596B1 (en) | 2010-06-23 | 2020-12-30 | CuRNA, Inc. | Treatment of sodium channel, voltage-gated, alpha subunit (scna) related diseases by inhibition of natural antisense transcript to scna |
US20130122046A1 (en) | 2010-07-09 | 2013-05-16 | Institut Pasteur Of Shanghai, Cas | Regulatory factor of foxp3 and regulatory t cells and use thereof |
TW201209163A (en) | 2010-07-12 | 2012-03-01 | Opko Curna Llc | Treatment of BCL2 binding component 3 (BBC3) related diseases by inhibition of natural antisense transcript to BBC3 |
RU2611190C2 (ru) | 2010-07-14 | 2017-02-21 | Курна, Инк. | Лечение заболеваний, связанных с геном dlg, путем ингибирования природного антисмыслового транскрипта гена dlg |
US20140011860A1 (en) * | 2010-07-19 | 2014-01-09 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals |
CN106434648A (zh) | 2010-07-19 | 2017-02-22 | F·C·贝内特 | 肌强直性营养障碍蛋白激酶(dmpk)表达的调节 |
WO2012018881A2 (en) | 2010-08-03 | 2012-02-09 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for the regulation of rna |
WO2012024478A2 (en) | 2010-08-19 | 2012-02-23 | Opko Curna Llc | Treatment of nicotinamide phosphoribosyltransferase (nampt) related diseases by inhibition of natural antisense transcript to nampt |
DK2614369T3 (da) | 2010-09-10 | 2016-05-02 | Epizyme Inc | Fremgangsmåde til bestemmelse af egnetheden for inhibitorer af human ezh2 i behandling |
EP2625274B1 (en) | 2010-10-06 | 2017-07-19 | CuRNA, Inc. | Treatment of sialidase 4 (neu4) related diseases by inhibition of natural antisense transcript to neu4 |
EP2630241B1 (en) | 2010-10-22 | 2018-10-17 | CuRNA, Inc. | Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua |
WO2012058268A2 (en) | 2010-10-27 | 2012-05-03 | Opko Curna Llc | Treatment of interferon-related developmental regulator 1 (ifrd1) related diseases by inhibition of natural antisense transcript to ifrd1 |
US9714427B2 (en) | 2010-11-11 | 2017-07-25 | The University Of North Carolina At Chapel Hill | Methods and compositions for unsilencing imprinted genes |
US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
AU2011325956B2 (en) | 2010-11-12 | 2016-07-14 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
US10000752B2 (en) | 2010-11-18 | 2018-06-19 | Curna, Inc. | Antagonat compositions and methods of use |
WO2012071238A2 (en) | 2010-11-23 | 2012-05-31 | Opko Curna Llc | Treatment of nanog related diseases by inhibition of natural antisense transcript to nanog |
DK2655621T3 (da) | 2010-12-20 | 2018-08-13 | Massachusetts Gen Hospital | Polycomb-associerede ikke-kodende RNAS |
WO2012109476A2 (en) | 2011-02-09 | 2012-08-16 | The University Of Rochester | Methods and compositions related to staufen 1 binding sites formed by duplexing alu elements |
WO2012144220A1 (en) | 2011-04-22 | 2012-10-26 | Oncotherapy Science, Inc. | Ezh2 as target gene for cancer therapy and diagnosis |
US8557787B2 (en) | 2011-05-13 | 2013-10-15 | The Board Of Trustees Of The Leland Stanford Junior University | Diagnostic, prognostic and therapeutic uses of long non-coding RNAs for cancer and regenerative medicine |
CA2838588C (en) | 2011-06-09 | 2021-09-14 | Curna, Inc. | Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn |
AU2012272518A1 (en) | 2011-06-24 | 2014-01-09 | Murdoch Childrens Research Institute | Treatment and diagnosis of epigenetic disorders and conditions |
WO2013006619A1 (en) | 2011-07-05 | 2013-01-10 | The General Hospital Corporation | Rna-yy1 interactions |
WO2013036403A1 (en) | 2011-09-06 | 2013-03-14 | Curna, Inc. | TREATMENT OF DISEASES RELATED TO ALPHA SUBUNITS OF SODIUM CHANNELS, VOLTAGE-GATED (SCNxA) WITH SMALL MOLECULES |
WO2013040429A1 (en) | 2011-09-14 | 2013-03-21 | Rana Therapeutics Inc. | Multimeric oligonucleotide compounds |
US9209196B2 (en) | 2011-11-30 | 2015-12-08 | Sharp Kabushiki Kaisha | Memory circuit, method of driving the same, nonvolatile storage device using the same, and liquid crystal display device |
ITMI20120275A1 (it) | 2012-02-24 | 2013-08-25 | Biogenera Societa A Responsabilita Limitata | Oligonucleotidi per la modulazione dell'espressione genica e loro usi |
US20150031750A1 (en) | 2012-03-15 | 2015-01-29 | The Scripps Research Institute | Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf |
CA2873809A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
WO2013173635A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
AP2014008100A0 (en) | 2012-05-16 | 2014-12-31 | Gen Hospital Corp | Compositions and methods for modulating hemoglobingene family expression |
US20150141320A1 (en) | 2012-05-16 | 2015-05-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | 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 |
EA201492119A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии apoa1 и abca1 |
WO2013173605A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating pten expression |
CA2873766A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating atp2a2 expression |
US20150133529A1 (en) | 2012-05-16 | 2015-05-14 | Rana Therapeutics, Inc. | Compositions and methods for modulating bdnf expression |
EP2850185A4 (en) | 2012-05-16 | 2015-12-30 | Rana Therapeutics Inc | COMPOSITIONS AND METHODS FOR MODULATING UTRN EXPRESSION |
US9567581B2 (en) | 2012-08-07 | 2017-02-14 | The General Hospital Corporation | Selective reactivation of genes on the inactive X chromosome |
CA2884608A1 (en) | 2012-09-14 | 2014-03-20 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
KR102112892B1 (ko) | 2012-11-15 | 2020-05-19 | 로슈 이노베이션 센터 코펜하겐 에이/에스 | 올리고뉴클레오티드 콘쥬게이트 |
WO2014172698A1 (en) | 2013-04-19 | 2014-10-23 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation nucleic acids through nonsense mediated decay |
AU2014274730A1 (en) | 2013-06-07 | 2016-01-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating FOXP3 expression |
US10190117B2 (en) | 2013-06-16 | 2019-01-29 | National University Corporation Tokyo Medical And Dental University | Double-stranded antisense nucleic acid with exon-skipping effect |
WO2015035476A1 (en) | 2013-09-16 | 2015-03-19 | University Of Western Sydney | Modulation of gene expression |
-
2013
- 2013-05-16 JP JP2015512858A patent/JP2015523854A/ja active Pending
- 2013-05-16 SG SG11201407483YA patent/SG11201407483YA/en unknown
- 2013-05-16 CN CN201380037632.9A patent/CN104540947A/zh active Pending
- 2013-05-16 EA EA201492123A patent/EA201492123A1/ru unknown
- 2013-05-16 AU AU2013262649A patent/AU2013262649A1/en not_active Abandoned
- 2013-05-16 CA CA2873794A patent/CA2873794A1/en not_active Abandoned
- 2013-05-16 KR KR1020147035185A patent/KR20150030205A/ko not_active Application Discontinuation
- 2013-05-16 DK DK13790819.0T patent/DK2850186T3/da active
- 2013-05-16 US US14/401,194 patent/US10059941B2/en active Active
- 2013-05-16 EP EP13790819.0A patent/EP2850186B1/en not_active Not-in-force
- 2013-05-16 WO PCT/US2013/041440 patent/WO2013173638A1/en active Application Filing
-
2014
- 2014-12-15 ZA ZA2014/09228A patent/ZA201409228B/en unknown
-
2015
- 2015-09-17 HK HK15109139.6A patent/HK1208700A1/xx unknown
Also Published As
Publication number | Publication date |
---|---|
ZA201409228B (en) | 2016-07-27 |
US10059941B2 (en) | 2018-08-28 |
KR20150030205A (ko) | 2015-03-19 |
EP2850186B1 (en) | 2018-12-19 |
CN104540947A (zh) | 2015-04-22 |
SG11201407483YA (en) | 2014-12-30 |
WO2013173638A1 (en) | 2013-11-21 |
AU2013262649A1 (en) | 2015-01-22 |
EP2850186A4 (en) | 2016-01-27 |
CA2873794A1 (en) | 2013-11-21 |
JP2015523854A (ja) | 2015-08-20 |
HK1208700A1 (en) | 2016-03-11 |
US20150252364A1 (en) | 2015-09-10 |
EA201492123A1 (ru) | 2015-10-30 |
EP2850186A1 (en) | 2015-03-25 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
DK2850186T3 (da) | Sammensætninger og fremgangsmåder til at modulere smn-genfamilie-ekspression | |
US11788089B2 (en) | Compositions and methods for modulating MECP2 expression | |
US10174328B2 (en) | Compositions and methods for treating amyotrophic lateral sclerosis | |
US10058623B2 (en) | Compositions and methods for modulating UTRN expression | |
US10174315B2 (en) | Compositions and methods for modulating hemoglobin gene family expression | |
US10174323B2 (en) | Compositions and methods for modulating ATP2A2 expression | |
US20150159161A1 (en) | Compositions and methods for modulating pten expression | |
US10837014B2 (en) | Compositions and methods for modulating SMN gene family expression | |
US20150191722A1 (en) | Compositions and methods for modulating apoa1 and abca1 expression | |
EP2849800A1 (en) | Compositions and methods for modulating bdnf expression | |
AU2014274730A1 (en) | Compositions and methods for modulating FOXP3 expression |