AR106311A1 - OLIGONUCLEOTID COMPOSITIONS AND THEIR METHODS - Google Patents
OLIGONUCLEOTID COMPOSITIONS AND THEIR METHODSInfo
- Publication number
- AR106311A1 AR106311A1 ARP160103093A ARP160103093A AR106311A1 AR 106311 A1 AR106311 A1 AR 106311A1 AR P160103093 A ARP160103093 A AR P160103093A AR P160103093 A ARP160103093 A AR P160103093A AR 106311 A1 AR106311 A1 AR 106311A1
- Authority
- AR
- Argentina
- Prior art keywords
- oligonucleotide
- composition
- oligonucleotides
- compositions provided
- transcript
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 14
- 238000000034 method Methods 0.000 title abstract 4
- 108091034117 Oligonucleotide Proteins 0.000 abstract 14
- 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 4
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 abstract 1
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 231100000053 low toxicity Toxicity 0.000 abstract 1
- 238000012986 modification Methods 0.000 abstract 1
- 230000004048 modification Effects 0.000 abstract 1
- 229910052698 phosphorus Inorganic materials 0.000 abstract 1
- 239000011574 phosphorus Substances 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Landscapes
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente se refiere a oligonucleótidos, composiciones diseñados y sus métodos. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas suministran un empalme alterado de un transcripto. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas tienen baja toxicidad. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas suministran mejores perfiles de ligación de proteínas. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas tienen mejor suministro. En algunas formas de realización, las composiciones oligonucleotídicas proporcionadas tienen mejor captación. En algunas formas de realización, la presente proporciona métodos para el tratamiento de enfermedades usando composiciones proporcionadas oligonucleotídicas. Reivindicación 1: Una composición oligonucleotídica, que comprende una primera pluralidad de oligonucleótidos de un tipo de oligonucleótido particular definido por: 1) secuencia de bases; 2) patrón de ligaciones estructurales; 3) patrón de centros quirales estructurales; y 4) patrón de modificaciones de fósforo estructurales, cuya composición es quiralmente controlada porque está enriquecida, respecto de una preparación sustancialmente racémica de oligonucleótidos que tienen la misma secuencia de bases, para oligonucleótidos del tipo de oligonucleótido particular, en donde: la composición oligonucleotídica que se caracteriza porque, cuando se pone en contacto con un transcripto en un sistema de empalme de transcriptos, el empalme del transcripto se altera respecto del observado en condiciones de referencia seleccionadas del grupo que consiste en ausencia de la composición, presencia de una composición de referencia y combinaciones de ellas. Reivindicación 42: Un método para el tratamiento de distrofia muscular de Duchenne, que comprende la administración a un sujeto susceptible o que padece de ella de una composición de acuerdo con cualquiera de las reivindicaciones 1 - 35 ó 40.This refers to oligonucleotides, designed compositions and their methods. In some embodiments, the oligonucleotide compositions provided provide an altered splice of a transcript. In some embodiments, the oligonucleotide compositions provided have low toxicity. In some embodiments, the oligonucleotide compositions provided provide better protein ligation profiles. In some embodiments, the oligonucleotide compositions provided have better delivery. In some embodiments, the oligonucleotide compositions provided have better uptake. In some embodiments, the present provides methods for the treatment of diseases using provided oligonucleotide compositions. Claim 1: An oligonucleotide composition, comprising a first plurality of oligonucleotides of a particular type of oligonucleotide defined by: 1) base sequence; 2) pattern of structural linkages; 3) pattern of structural chiral centers; and 4) pattern of structural phosphorus modifications, whose composition is chirally controlled because it is enriched, relative to a substantially racemic preparation of oligonucleotides having the same base sequence, for oligonucleotides of the particular oligonucleotide type, wherein: the oligonucleotide composition that It is characterized in that, when a transcript is contacted in a transcript splice system, the transcript splice is altered with respect to that observed in reference conditions selected from the group consisting of absence of the composition, presence of a reference composition and combinations of them. Claim 42: A method for the treatment of Duchenne muscular dystrophy, comprising administration to a susceptible or suffering subject of a composition according to any one of claims 1-35 or 40.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562239839P | 2015-10-09 | 2015-10-09 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR106311A1 true AR106311A1 (en) | 2018-01-03 |
Family
ID=61158543
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP160103093A AR106311A1 (en) | 2015-10-09 | 2016-10-07 | OLIGONUCLEOTID COMPOSITIONS AND THEIR METHODS |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR106311A1 (en) |
-
2016
- 2016-10-07 AR ARP160103093A patent/AR106311A1/en unknown
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| FB | Suspension of granting procedure |