AR127137A2 - SERPINC1 ARNI COMPOSITIONS AND THEIR METHODS OF USE - Google Patents
SERPINC1 ARNI COMPOSITIONS AND THEIR METHODS OF USEInfo
- Publication number
- AR127137A2 AR127137A2 ARP220102569A ARP220102569A AR127137A2 AR 127137 A2 AR127137 A2 AR 127137A2 AR P220102569 A ARP220102569 A AR P220102569A AR P220102569 A ARP220102569 A AR P220102569A AR 127137 A2 AR127137 A2 AR 127137A2
- Authority
- AR
- Argentina
- Prior art keywords
- serpinc1
- dsrna
- inhibiting
- expression
- cell
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 5
- 239000000203 mixture Substances 0.000 title abstract 3
- 102100022977 Antithrombin-III Human genes 0.000 title 1
- 241000320892 Clerodendrum phlomidis Species 0.000 title 1
- 101000757319 Homo sapiens Antithrombin-III Proteins 0.000 title 1
- 229940125365 angiotensin receptor blocker-neprilysin inhibitor Drugs 0.000 title 1
- 239000002773 nucleotide Substances 0.000 abstract 8
- 125000003729 nucleotide group Chemical group 0.000 abstract 8
- 230000002401 inhibitory effect Effects 0.000 abstract 6
- 230000000692 anti-sense effect Effects 0.000 abstract 5
- 101150026876 SERPINC1 gene Proteins 0.000 abstract 4
- 229920002477 rna polymer Polymers 0.000 abstract 3
- 108091030071 RNAI Proteins 0.000 abstract 2
- 230000009368 gene silencing by RNA Effects 0.000 abstract 2
- 208000031220 Hemophilia Diseases 0.000 abstract 1
- 208000009292 Hemophilia A Diseases 0.000 abstract 1
- 230000015556 catabolic process Effects 0.000 abstract 1
- 238000006731 degradation reaction Methods 0.000 abstract 1
- 208000031169 hemorrhagic disease Diseases 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
Landscapes
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Composiciones de ARNi, por ejemplo, ácido ribonucleico de cadena doble (ARNcd), dirigido al gen Serpinc1, y métodos de uso de dicho ARNi, por ejemplo, ARNcd, composiciones para inhibir la expresión de Serpinc1 y métodos de tratamiento de sujetos que sufren de un trastorno de sangrado, tal como hemofilia. Reivindicación 1: Un ácido ribonucleico de cadena doble (ARNcd) para inhibir la expresión de Serpinc1, caracterizado porque dicho ARNcd comprende una cadena orientada en el sentido del marco de lectura y una cadena antisentido, en donde dicha cadena orientada en el sentido del marco de lectura comprende al menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de la secuencia de nucleótidos de la SEQ ID Nº 1 y dicha cadena antisentido comprende al menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de la secuencia de nucleótidos de la SEQ ID Nº 5. Reivindicación 2: Un ácido ribonucleico de cadena doble (ARNcd) para inhibir la expresión de Serpinc1, caracterizado porque dicho ARNcd comprende una cadena orientada en el sentido del marco de lectura y una cadena antisentido, donde la cadena antisentido comprende una región de complementariedad que comprende al menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de cualquiera de las secuencias antisentido enumeradas en cualquiera de las Tablas 3, 4, 8, 11, 12, 14, 15, 20 y 21. Reivindicación 25: Una célula, caracterizada porque comprende al vector de la Reivindicación 22. Reivindicación 26: Una composición farmacéutica para inhibir la expresión de un gen Serpinc1, caracterizada porque comprende al ARNcd de la Reivindicación 1 ó 2 o al vector de la Reivindicación 22. Reivindicación 27: Un método de inhibición de la expresión de Serpinc1 en una célula, caracterizado porque dicho método comprende: (a) poner la célula en contacto con el ARNcd de la Reivindicación 1 ó 2 o con el vector de la Reivindicación 22; y (b) mantener la célula producida en el paso (a) por un tiempo suficiente como para obtener la degradación del transcripto de ARNm de un gen Serpinc1, inhibiendo de esa manera la expresión del gen Serpinc1 en la célula.RNAi compositions, e.g., double-stranded ribonucleic acid (dsRNA), targeting the Serpinc1 gene, and methods of using said RNAi, e.g., dsRNA, compositions for inhibiting the expression of Serpinc1, and methods of treating subjects suffering from a bleeding disorder, such as hemophilia. Claim 1: A double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of Serpinc1, characterized in that said dsRNA comprises a frame-oriented strand and an antisense strand, wherein said frame-oriented strand read comprises at least 15 contiguous nucleotides that differ by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO: 1 and said antisense strand comprises at least 15 contiguous nucleotides that differ by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO: 5. Claim 2: A double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of Serpinc1, characterized in that said dsRNA comprises a strand oriented in the sense of the reading frame and an antisense strand, where the antisense strand comprises a region of complementarity comprising at least 15 contiguous nucleotides that differ by no more than 3 nucleotides from any of the antisense sequences listed in any of Tables 3, 4, 8, 11, 12, 14, 15, 20 and 21. Claim 25: A cell, characterized in that it comprises the vector of Claim 22. Claim 26: A pharmaceutical composition for inhibiting the expression of a Serpinc1 gene, characterized in that it comprises the dsRNA of Claim 1 or 2 or the vector of Claim 22. Claim 27: A method of inhibiting the expression of Serpinc1 in a cell, characterized in that said method comprises: (a) putting the cell in contact with the dsRNA of Claim 1 or 2 or with the vector of Claim 22; and (b) maintaining the cell produced in step (a) for a time sufficient to obtain degradation of the mRNA transcript of a Serpinc1 gene, thereby inhibiting expression of the Serpinc1 gene in the cell.
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261638952P | 2012-04-26 | 2012-04-26 | |
| US202013837129A | 2020-03-15 | 2020-03-15 | |
| US202061669249P | 2020-07-09 | 2020-07-09 | |
| US202061734573P | 2020-12-07 | 2020-12-07 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR127137A2 true AR127137A2 (en) | 2023-12-20 |
Family
ID=89309883
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220102569A AR127137A2 (en) | 2012-04-26 | 2022-09-23 | SERPINC1 ARNI COMPOSITIONS AND THEIR METHODS OF USE |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR127137A2 (en) |
-
2022
- 2022-09-23 AR ARP220102569A patent/AR127137A2/en unknown
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