AR097920A1 - COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GENE SINTASA 1 OF 5-AMINOLEVULIN ACID (ALAS1) - Google Patents
COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GENE SINTASA 1 OF 5-AMINOLEVULIN ACID (ALAS1)Info
- Publication number
- AR097920A1 AR097920A1 ARP140103699A ARP140103699A AR097920A1 AR 097920 A1 AR097920 A1 AR 097920A1 AR P140103699 A ARP140103699 A AR P140103699A AR P140103699 A ARP140103699 A AR P140103699A AR 097920 A1 AR097920 A1 AR 097920A1
- Authority
- AR
- Argentina
- Prior art keywords
- alas1
- expression
- compositions
- dsrna
- inhibit
- Prior art date
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- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Abstract
Se refiere a composiciones de ácido ribonucleico bicatenario (dsARN) opcionalmente conjugado a un ligando tal como N-acetilgalactosamina. Los dsARN están dirigidos al gen ALAS1. Métodos de uso de tales composiciones de dsARN para alterar (por ejemplo, inhibir) la expresión de ALAS1, tal como tratamientos de sujetos con porfiria. Reivindicación 1: Un ácido ribonucleico bicatenario (dsARN) para inhibir la expresión de ALAS1, en donde dicho dsARN comprende una cadena con sentido y una cadena antisentido, donde la cadena antisentido comprende una región de complementariedad con un transcripto de ARN de ALAS1 (por ejemplo, SEQ ID N º1), cuya cadena antisentido comprende al menos 20 nucleótidos contiguos de la secuencia antisentido de UAAGAUGAGACACUCUUUCUGGU (SEQ ID Nº 4153) o UAAGAUGAGACACUCTUUCUGGU (SEQ ID Nº 4154).It refers to double stranded ribonucleic acid (dsRNA) compositions optionally conjugated to a ligand such as N-acetylgalactosamine. The dsRNAs are directed to the ALAS1 gene. Methods of using such dsRNA compositions to alter (for example, inhibit) the expression of ALAS1, such as treatments of subjects with porphyria. Claim 1: A double stranded ribonucleic acid (dsRNA) for inhibiting the expression of ALAS1, wherein said dsRNA comprises a sense chain and an antisense chain, wherein the antisense chain comprises a region of complementarity with an ALAS1 RNA transcript (for example , SEQ ID No. 1), whose antisense chain comprises at least 20 contiguous nucleotides of the antisense sequence of UAAGAUGAGACACUCUUUCUGGU (SEQ ID No. 4153) or UAAGAUGAGACACUCTUUCUGGU (SEQ ID No. 4154).
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361887288P | 2013-10-04 | 2013-10-04 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR097920A1 true AR097920A1 (en) | 2016-04-20 |
Family
ID=58698725
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP140103699A AR097920A1 (en) | 2013-10-04 | 2014-10-03 | COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GENE SINTASA 1 OF 5-AMINOLEVULIN ACID (ALAS1) |
| ARP200101035A AR118658A2 (en) | 2013-10-04 | 2020-04-13 | COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF THE ALAS1 GENE |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP200101035A AR118658A2 (en) | 2013-10-04 | 2020-04-13 | COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF THE ALAS1 GENE |
Country Status (2)
| Country | Link |
|---|---|
| AR (2) | AR097920A1 (en) |
| MA (1) | MA39000B1 (en) |
-
2014
- 2014-10-03 AR ARP140103699A patent/AR097920A1/en active IP Right Grant
- 2014-10-03 MA MA39000A patent/MA39000B1/en unknown
-
2020
- 2020-04-13 AR ARP200101035A patent/AR118658A2/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| MA39000A1 (en) | 2018-05-31 |
| AR118658A2 (en) | 2021-10-20 |
| MA39000B1 (en) | 2018-12-31 |
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|---|---|---|---|
| FG | Grant, registration |