Pharmaceutical compositions and treatment of genetic diseases associated with nonsense mediated RNA decay
US-9216180-B2 · Dec 22, 2015 · US
US2016287560A1 · US · A1
| Field | Value |
|---|---|
| Publication number | US-2016287560-A1 |
| Application number | US-201615182001-A |
| Country | US |
| Kind code | A1 |
| Filing date | Jun 14, 2016 |
| Priority date | Apr 29, 2003 |
| Publication date | Oct 6, 2016 |
| Grant date | — |
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Invented is a method of treating degenerative diseases/injuries, in a mammal, including a human, in need thereof which comprises the administration of a therapeutically effective amount of a non-peptide TPO receptor agonist to such mammal.
Opening claim text (preview).
What is claimed is: 1 . An in vitro or ex vivo method of enhancing the differentation of t lood components in human fetal cord blood into functional cells which method comprises the addition of an effective amount of a non-peptide TPO receptor agonist selected from: 3′-{N′-[1-(3,4-Dimethylphenyl)-3-methyl-5-oxo-1,5-dihydropyrazol-4-ylidene]hydrazino}-2′-hydroxybiphenyl-3-carboxylic acid, or a pharmaceutically acceptable salt thereof, and 3-{N′-[1-(3,4-dimethylphenyl)-3-methyl-5-oxo-1,5-dihydropyrazol-4-ylidene]hydrazino}-2-hydroxy-3′-tetrazol-5-ylbiphenyl, or a pharmaceutically acceptable salt thereof; to a culture medium containing human fetal cord blood; followed by optional isolation of the functional cells. 2 . The method of claim 1 wherein progenitor cells are enhanced. 3 . A method of transfusing human fetal cord blood which method comprises the addition of an effective amount of a non-peptide TPO receptor agonist selected from: 3′-{N′-[1-(3,4-Dimethylphenyl)-3-methyl-5-oxo-1,5-dihydropyrazol-4-ylidene]hydrazino}-2′-hydroxybiphenyl-3-carboxylic acid, or a pharmaceutically acceptable salt thereof, and 3-{N′-[1-(3,4-dimethylphenyl)-3-methyl-5-oxo-1,5-dihydropyrazol-4-ylidene]hydrazino}-2-hydroxy-3′-tetrazol-5-ylbiphenyl, or a pharmaceutically acceptable salt thereof; to a patient receiving human fetal cord blood.
1,2-Diazoles · CPC title
Vertebrate cells · CPC title
having oxo groups directly attached to the heterocyclic ring, e.g. antipyrine, phenylbutazone, sulfinpyrazone · CPC title
Hepatocytes · CPC title
having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole · CPC title
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