AR105509A1 - XANTINA DEHYDROGENASE (XDH) RNAi COMPOSITIONS AND SAME USE METHODS - Google Patents
XANTINA DEHYDROGENASE (XDH) RNAi COMPOSITIONS AND SAME USE METHODSInfo
- Publication number
- AR105509A1 AR105509A1 ARP160102290A ARP160102290A AR105509A1 AR 105509 A1 AR105509 A1 AR 105509A1 AR P160102290 A ARP160102290 A AR P160102290A AR P160102290 A ARP160102290 A AR P160102290A AR 105509 A1 AR105509 A1 AR 105509A1
- Authority
- AR
- Argentina
- Prior art keywords
- xdh
- ligand
- mrna agent
- dehydrogenase
- gene
- Prior art date
Links
- 108091030071 RNAI Proteins 0.000 title abstract 4
- 230000009368 gene silencing by RNA Effects 0.000 title abstract 4
- 101710088194 Dehydrogenase Proteins 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 239000003795 chemical substances by application Substances 0.000 abstract 8
- 239000002773 nucleotide Substances 0.000 abstract 6
- 125000003729 nucleotide group Chemical group 0.000 abstract 6
- 108010091383 Xanthine dehydrogenase Proteins 0.000 abstract 5
- 102000005773 Xanthine dehydrogenase Human genes 0.000 abstract 5
- 108010093894 Xanthine oxidase Proteins 0.000 abstract 5
- 108020004999 messenger RNA Proteins 0.000 abstract 5
- 239000003446 ligand Substances 0.000 abstract 4
- 108091081021 Sense strand Proteins 0.000 abstract 3
- 230000000692 anti-sense effect Effects 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical class CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 abstract 1
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 abstract 1
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 abstract 1
- 101150065223 XDH gene Proteins 0.000 abstract 1
- 150000001875 compounds Chemical class 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 229920002477 rna polymer Polymers 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
Landscapes
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Agentes de ARNi, por ejemplo, agentes de ARNi de hebra doble, dirigidos a un gen de una xantina deshidrogenasa (XDH), y métodos de uso de dichos agentes de ARNi de hebra doble para inhibir la expresión de un gen de XDH y métodos de tratamiento de sujetos qua sufren de una enfermedad asociada XDH. Reivindicación 1: Un ácido ribonucleico de hebra doble (ARNhd) para inhibir la expresión de un gen de la xantina deshidrogenasa (XDH), caracterizado porque dicho ARNhd comprende una hebra sentido y una hebra antisentido, en donde dicha hebra sentido comprende por lo menos 15 nucleótidos contiguos que no difieren en más de 3 nucleótidos de la secuencia de nucleótidos de la SEQ ID Nº 1 y dicha hebra antisentido comprende por lo menos 15 nucleótidos contiguos que no difieren en más de 3 nucleótidos de la secuencia de nucleótidos de la SEQ ID Nº 2. Reivindicación 19: El agente de ARNhd de cualquiera de las reivindicaciones 1 - 4, caracterizado porque además comprende un ligando. Reivindicación 20: El agente de ARNhd de la reivindicación 19, caracterizado porque el ligando está conjugado al extremo 3 de la hebra sentido de dicho agente de ARNhd. Reivindicación 21: El agente de ARNhd de la reivindicación 8, caracterizado porque el ligando es un derivado de N-acetilgalactosamina (GalNAc). Reivindicación 22: El agente de ARNhd de la reivindicación 21, caracterizado porque el ligando es un compuesto de fórmula (1).RNAi agents, for example, double stranded RNAi agents, targeting a gene of a xanthine dehydrogenase (XDH), and methods of using said double stranded RNAi agents to inhibit the expression of an XDH gene and methods of Treatment of subjects who suffer from an XDH associated disease. Claim 1: A double stranded ribonucleic acid (hRNA) for inhibiting the expression of a xanthine dehydrogenase (XDH) gene, characterized in that said hRNA comprises a sense strand and an antisense strand, wherein said sense strand comprises at least 15 contiguous nucleotides that do not differ by more than 3 nucleotides from the nucleotide sequence of SEQ ID No. 1 and said antisense strand comprises at least 15 contiguous nucleotides that do not differ by more than 3 nucleotides from the nucleotide sequence of SEQ ID No. 2. Claim 19: The mRNA agent of any of claims 1-4, characterized in that it further comprises a ligand. Claim 20: The mRNA agent of claim 19, characterized in that the ligand is conjugated to the 3-terminus of the sense strand of said mRNA agent. Claim 21: The mRNA agent of claim 8, characterized in that the ligand is a derivative of N-acetylgalactosamine (GalNAc). Claim 22: The mRNA agent of claim 21, characterized in that the ligand is a compound of formula (1).
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562197221P | 2015-07-27 | 2015-07-27 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR105509A1 true AR105509A1 (en) | 2017-10-11 |
Family
ID=60244681
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP160102290A AR105509A1 (en) | 2015-07-27 | 2016-07-27 | XANTINA DEHYDROGENASE (XDH) RNAi COMPOSITIONS AND SAME USE METHODS |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR105509A1 (en) |
-
2016
- 2016-07-27 AR ARP160102290A patent/AR105509A1/en unknown
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FB | Suspension of granting procedure |