BR112014025020A2 - composições e métodos para inibição da expressão do gene alas1 - Google Patents
composições e métodos para inibição da expressão do gene alas1Info
- Publication number
- BR112014025020A2 BR112014025020A2 BR112014025020A BR112014025020A BR112014025020A2 BR 112014025020 A2 BR112014025020 A2 BR 112014025020A2 BR 112014025020 A BR112014025020 A BR 112014025020A BR 112014025020 A BR112014025020 A BR 112014025020A BR 112014025020 A2 BR112014025020 A2 BR 112014025020A2
- Authority
- BR
- Brazil
- Prior art keywords
- compositions
- methods
- alas1 gene
- gene expression
- dsrna
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 6
- 230000014509 gene expression Effects 0.000 title abstract 4
- 230000002401 inhibitory effect Effects 0.000 title abstract 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 4
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 229920002477 rna polymer Polymers 0.000 abstract 2
- 230000008685 targeting Effects 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
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- 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
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
- A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
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- 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
- C12N15/1137—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 enzymes
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering N.A.
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- C12N2310/31—Chemical structure of the backbone
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/321—2'-O-R Modification
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- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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- C12N2310/3521—Methyl
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- C12N2310/53—Physical structure partially self-complementary or closed
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- C12Y203/00—Acyltransferases (2.3)
- C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
- C12Y203/01037—5-Aminolevulinate synthase (2.3.1.37)
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Abstract
abstract the invention relates to double-stranded ribonucleic acid (dsrna) compositions targeting the alas1 gene, and methods of using such dsrna compositions to alter (e.g., inhibit) expression of alas1. ___________________________________________________________________ resumo patente de invenção: "composições e métodos para inibição da expressão do gene alas1". a invenção se relaciona com composições de ácido ribonucleico de fita dupla (dsrna) se dirigindo ao gene alas1, e métodos de uso de tais composições de dsrna para alterar (p.ex., inibir) a expressão de alas1.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261622288P | 2012-04-10 | 2012-04-10 | |
US13/835,613 US9133461B2 (en) | 2012-04-10 | 2013-03-15 | Compositions and methods for inhibiting expression of the ALAS1 gene |
PCT/US2013/036006 WO2013155204A2 (en) | 2012-04-10 | 2013-04-10 | Compositions and methods for inhibiting expression of the alas1 gene |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112014025020A2 true BR112014025020A2 (pt) | 2017-07-11 |
Family
ID=48326393
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112014025020A BR112014025020A2 (pt) | 2012-04-10 | 2013-04-10 | composições e métodos para inibição da expressão do gene alas1 |
Country Status (28)
Country | Link |
---|---|
US (8) | US9133461B2 (pt) |
EP (3) | EP3868883A1 (pt) |
JP (4) | JP6422854B2 (pt) |
KR (3) | KR102139451B1 (pt) |
CN (2) | CN104540948B (pt) |
AR (1) | AR090641A1 (pt) |
AU (4) | AU2013245949A1 (pt) |
BR (1) | BR112014025020A2 (pt) |
CA (2) | CA3169218A1 (pt) |
CL (2) | CL2014002725A1 (pt) |
CO (1) | CO7121351A2 (pt) |
CY (1) | CY1119491T1 (pt) |
DK (1) | DK2836595T3 (pt) |
ES (1) | ES2640260T3 (pt) |
HK (2) | HK1201876A1 (pt) |
HR (1) | HRP20171369T1 (pt) |
HU (1) | HUE034311T2 (pt) |
IL (5) | IL281295B (pt) |
LT (1) | LT2836595T (pt) |
ME (1) | ME02850B (pt) |
MX (3) | MX368723B (pt) |
PL (1) | PL2836595T3 (pt) |
PT (1) | PT2836595T (pt) |
RS (1) | RS56428B1 (pt) |
RU (2) | RU2019111696A (pt) |
SI (1) | SI2836595T1 (pt) |
WO (1) | WO2013155204A2 (pt) |
ZA (1) | ZA201408111B (pt) |
Families Citing this family (29)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9133461B2 (en) | 2012-04-10 | 2015-09-15 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the ALAS1 gene |
WO2014179625A1 (en) * | 2013-05-01 | 2014-11-06 | Isis Pharmaceuticals, Inc. | COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN (a) EXPRESSION |
KR102365486B1 (ko) | 2013-08-28 | 2022-02-18 | 아이오니스 파마수티컬즈, 인코포레이티드 | 프리칼리크레인 (pkk) 발현의 조절 |
BR122020001264B1 (pt) * | 2013-10-04 | 2022-11-22 | Icahn School Of Medicine At Mount Sinai | Ácido ribonucleico de fita dupla (dsrna) para inibição da expressão de alas1, composição farmacêutica para inibição da expressão de um gene alas1, métodos de inibição da expressão de alas1 em uma célula e para diminuir um nível de uma porfirina ou de um precursor de porfirina em uma célula, usos de um dsrna, método para avaliar o nível de mrna de alas1 extracelular em circulação em um indivíduo, composição e uso da mesma |
EP3060664B1 (en) * | 2013-10-25 | 2021-07-07 | Sanofi | Microrna compounds and methods for modulating mir-21 activity |
JP6886818B2 (ja) | 2013-12-27 | 2021-06-16 | ダイセルナ ファーマシューティカルズ, インコーポレイテッドDicerna Pharmaceuticals, Inc. | 二本鎖rnaによるグリコール酸オキシダーゼ(hao1)の特異的阻害に関する方法および組成物 |
EP3105331B1 (en) | 2014-02-11 | 2021-06-23 | Alnylam Pharmaceuticals, Inc. | Ketohexokinase (khk) irna compositions and methods of use thereof |
CA2946003A1 (en) | 2014-05-01 | 2015-11-05 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating angiopoietin-like 3 expression |
DK3137091T3 (da) * | 2014-05-01 | 2021-01-25 | Ionis Pharmaceuticals Inc | Konjugater af modificerede antisense-oligonukleotider og anvendelse deraf til modulation af pkk-ekspression |
EP3757215A3 (en) * | 2014-05-01 | 2021-03-17 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating growth hormone receptor expression |
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