BR112022006207A2 - Oligonucleotídeo, composição de oligonucleotídeo, composição farmacêutica, método, método para diminuir a atividade, expressão e/ou nível de um gene alvo ou seu produto de gene em uma célula, método para reduzir focos em uma população de células, método para reduzir o nível de uma proteína de repetição de dipeptídeo (dpr), método para knockdown preferencial de uma transcrição de rna, e método para preparar um oligonucleotídeo ou composição - Google Patents
Oligonucleotídeo, composição de oligonucleotídeo, composição farmacêutica, método, método para diminuir a atividade, expressão e/ou nível de um gene alvo ou seu produto de gene em uma célula, método para reduzir focos em uma população de células, método para reduzir o nível de uma proteína de repetição de dipeptídeo (dpr), método para knockdown preferencial de uma transcrição de rna, e método para preparar um oligonucleotídeo ou composiçãoInfo
- Publication number
- BR112022006207A2 BR112022006207A2 BR112022006207A BR112022006207A BR112022006207A2 BR 112022006207 A2 BR112022006207 A2 BR 112022006207A2 BR 112022006207 A BR112022006207 A BR 112022006207A BR 112022006207 A BR112022006207 A BR 112022006207A BR 112022006207 A2 BR112022006207 A2 BR 112022006207A2
- Authority
- BR
- Brazil
- Prior art keywords
- oligonucleotide
- composition
- level
- reduce
- cell
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 14
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 7
- 108090000623 proteins and genes Proteins 0.000 title abstract 6
- 239000000203 mixture Substances 0.000 title abstract 5
- 108010016626 Dipeptides Proteins 0.000 title abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 title abstract 2
- 102000004169 proteins and genes Human genes 0.000 title abstract 2
- 238000013518 transcription Methods 0.000 title abstract 2
- 230000035897 transcription Effects 0.000 title abstract 2
- 102000043334 C9orf72 Human genes 0.000 abstract 2
- 108700030955 C9orf72 Proteins 0.000 abstract 2
- 101150014718 C9orf72 gene Proteins 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 201000011240 Frontotemporal dementia Diseases 0.000 abstract 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 abstract 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
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
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Neurosurgery (AREA)
- Public Health (AREA)
- Neurology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hospice & Palliative Care (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Abstract
OLIGONUCLEOTÍDEO, COMPOSIÇÃO DE OLIGONUCLEOTÍDEO, COMPOSIÇÃO FARMACÊUTICA, MÉTODO, MÉTODO PARA DIMINUIR A ATIVIDADE, EXPRESSÃO E/OU NÍVEL DE UM GENE ALVO OU SEU PRODUTO DE GENE EM UMA CÉLULA, MÉTODO PARA REDUZIR FOCOS EM UMA POPULAÇÃO DE CÉLULAS, MÉTODO PARA REDUZIR O NÍVEL DE UMA PROTEÍNA DE REPETIÇÃO DE DIPEPTÍDEO (DPR), MÉTODO PARA KNOCKDOWN PREFERENCIAL DE UMA TRANSCRIÇÃO DE RNA, E MÉTODO PARA PREPARAR UM OLIGONUCLEOTÍDEO OU COMPOSIÇÃO. Entre outros aspectos, a presente revelação fornece oligonucleotídeos C9orf72, composições e métodos de uso dos mesmos. Em algumas modalidades, a presente revelação fornece métodos para tratar afecções, distúrbios ou doenças associados a C9orf72, tais como esclerose lateral amiotrófica e demência frontotemporal.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962911340P | 2019-10-06 | 2019-10-06 | |
| US202062983736P | 2020-03-01 | 2020-03-01 | |
| PCT/US2020/032244 WO2020227691A2 (en) | 2019-05-09 | 2020-05-08 | Oligonucleotide compositions and methods of use thereof |
| US202063069704P | 2020-08-24 | 2020-08-24 | |
| PCT/US2020/054307 WO2021071788A2 (en) | 2019-10-06 | 2020-10-05 | Oligonucleotide compositions and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022006207A2 true BR112022006207A2 (pt) | 2022-07-26 |
Family
ID=75438309
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022006207A BR112022006207A2 (pt) | 2019-10-06 | 2020-10-05 | Oligonucleotídeo, composição de oligonucleotídeo, composição farmacêutica, método, método para diminuir a atividade, expressão e/ou nível de um gene alvo ou seu produto de gene em uma célula, método para reduzir focos em uma população de células, método para reduzir o nível de uma proteína de repetição de dipeptídeo (dpr), método para knockdown preferencial de uma transcrição de rna, e método para preparar um oligonucleotídeo ou composição |
Country Status (11)
| Country | Link |
|---|---|
| EP (1) | EP4037695A4 (pt) |
| JP (1) | JP2022550915A (pt) |
| KR (1) | KR20220070324A (pt) |
| CN (1) | CN114585370A (pt) |
| AU (1) | AU2020363344A1 (pt) |
| BR (1) | BR112022006207A2 (pt) |
| CA (1) | CA3156176A1 (pt) |
| IL (1) | IL291974A (pt) |
| MX (1) | MX2022004102A (pt) |
| TW (1) | TW202126810A (pt) |
| WO (1) | WO2021071788A2 (pt) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| CN104661664B (zh) | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| WO2017160741A1 (en) | 2016-03-13 | 2017-09-21 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| JP7296882B2 (ja) | 2016-11-23 | 2023-06-23 | ウェイブ ライフ サイエンシズ リミテッド | ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法 |
| CN110997692A (zh) | 2017-06-02 | 2020-04-10 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| JP7402696B2 (ja) | 2017-06-21 | 2023-12-21 | ウェイブ ライフ サイエンシズ リミテッド | 合成のための化合物、組成物、及び方法 |
| SG11202000274RA (en) | 2017-08-08 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| SG11202000276YA (en) | 2017-09-18 | 2020-04-29 | Wave Life Sciences Ltd | Technologies for oligonucleotide preparation |
| SG11202001783YA (en) | 2017-10-12 | 2020-03-30 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| WO2024013361A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2856403T3 (es) * | 2014-03-18 | 2021-09-27 | Univ Massachusetts | Composiciones a base de rAAV y procedimientos para el tratamiento de la esclerosis lateral amiotrofica |
| PL3283080T3 (pl) * | 2015-04-16 | 2020-07-27 | Ionis Pharmaceuticals, Inc. | Kompozycja do modulowania ekspresji c9orf72 |
| SG11202000274RA (en) * | 2017-08-08 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| WO2020227691A2 (en) * | 2019-05-09 | 2020-11-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
-
2020
- 2020-10-05 BR BR112022006207A patent/BR112022006207A2/pt not_active Application Discontinuation
- 2020-10-05 MX MX2022004102A patent/MX2022004102A/es unknown
- 2020-10-05 CN CN202080069266.5A patent/CN114585370A/zh active Pending
- 2020-10-05 AU AU2020363344A patent/AU2020363344A1/en active Pending
- 2020-10-05 KR KR1020227015125A patent/KR20220070324A/ko unknown
- 2020-10-05 WO PCT/US2020/054307 patent/WO2021071788A2/en active Application Filing
- 2020-10-05 IL IL291974A patent/IL291974A/en unknown
- 2020-10-05 JP JP2022520976A patent/JP2022550915A/ja active Pending
- 2020-10-05 EP EP20875598.3A patent/EP4037695A4/en active Pending
- 2020-10-05 CA CA3156176A patent/CA3156176A1/en active Pending
- 2020-10-06 TW TW109134610A patent/TW202126810A/zh unknown
Also Published As
| Publication number | Publication date |
|---|---|
| TW202126810A (zh) | 2021-07-16 |
| WO2021071788A2 (en) | 2021-04-15 |
| IL291974A (en) | 2022-06-01 |
| EP4037695A2 (en) | 2022-08-10 |
| AU2020363344A1 (en) | 2022-05-26 |
| MX2022004102A (es) | 2022-04-26 |
| CA3156176A1 (en) | 2021-04-15 |
| JP2022550915A (ja) | 2022-12-05 |
| EP4037695A4 (en) | 2024-03-27 |
| KR20220070324A (ko) | 2022-05-30 |
| CN114585370A (zh) | 2022-06-03 |
| WO2021071788A3 (en) | 2021-05-14 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| BR112022006207A2 (pt) | Oligonucleotídeo, composição de oligonucleotídeo, composição farmacêutica, método, método para diminuir a atividade, expressão e/ou nível de um gene alvo ou seu produto de gene em uma célula, método para reduzir focos em uma população de células, método para reduzir o nível de uma proteína de repetição de dipeptídeo (dpr), método para knockdown preferencial de uma transcrição de rna, e método para preparar um oligonucleotídeo ou composição | |
| Konovalova et al. | Interplay between MicroRNAs and oxidative stress in neurodegenerative diseases | |
| Duscher et al. | Aging disrupts cell subpopulation dynamics and diminishes the function of mesenchymal stem cells | |
| Inoue et al. | Bone protection by inhibition of microRNA-182 | |
| Decque et al. | Sumoylation coordinates the repression of inflammatory and anti-viral gene-expression programs during innate sensing | |
| BR112022006205A2 (pt) | Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou método | |
| Van Buul et al. | Mesenchymal stem cells secrete factors that inhibit inflammatory processes in short-term osteoarthritic synovium and cartilage explant culture | |
| MX2021012126A (es) | Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central. | |
| EP3500585A1 (en) | Nucleic acid products and methods of administration thereof | |
| EA202191313A1 (ru) | Композиции на основе липидных наночастиц | |
| Ryan et al. | Time-dependent changes in gene expression induced by secreted amyloid precursor protein-alpha in the rat hippocampus | |
| BR112020026125A8 (pt) | Compostos oligonucleotídeos superiores ou 5'-cap- trinucleotídeo e seus usos na estabilização de rna, expressão de proteínas e em terapia | |
| Moran-Salvador et al. | Fibrogenic activity of MECP2 is regulated by phosphorylation in hepatic stellate cells | |
| Kuzma-Kozakiewicz et al. | New therapeutic targets for amyotrophic lateral sclerosis | |
| Hwang et al. | Induction of pro-inflammatory cytokines by 29-kDa FN-f via cGAS/STING pathway | |
| PH12020551642A1 (en) | Aav compositions, methods of making and methods of use | |
| CO2019012484A2 (es) | Disminución de homocisteína mediada por enzimas humanas para el tratamiento de pacientes con hiperhomocisteinemia y homocistinuria | |
| Yang et al. | miR-146a down-regulation alleviates H 2 O 2-induced cytotoxicity of PC12 cells by regulating MCL1/JAK/STAT pathway: miR-146a down-regulation relieves H2O2-induced PC12 cells cytotoxicity by MCL1/JAK/STAT | |
| JP2022188178A (ja) | Ｍａｓｔ４遺伝子を利用した細胞外基質生産用組成物及びその製造方法 | |
| BR112021019109A2 (pt) | Célula rpe manipulada, composições, molécula isolada de dna de fita dupla, dispositivo implantável, cápsula de hidrogel, e, método para aplicar uma terapia com fvii | |
| Toh et al. | A report on the International Society for Cell & Gene Therapy 2022 Scientific Signature Series,“Therapeutic advances with native and engineered human extracellular vesicles” | |
| Chen et al. | Peimine suppresses interleukin‑1β‑induced inflammation via MAPK downregulation in chondrocytes | |
| Li et al. | Long intergenic non-coding RNA-00917 regulates the proliferation, inflammation, and pyroptosis of nucleus pulposus cells via targeting miR-149-5p/NOD-like receptor protein 1 axis | |
| Wa et al. | miRNA-140 inhibits C3H10T1/2 mesenchymal stem cell proliferation by targeting CXCL12 during transforming growth factor-β3-induced chondrogenic differentiation | |
| Walters et al. | Divergent functions of histone acetyltransferases KAT2A and KAT2B in keratinocyte self-renewal and differentiation |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B11A | Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing | ||
| B11Y | Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette] |