Inducers of gamma globin gene expression and screening assays therefor
US5700640A · kind A · utility
Assignee
Inventors
Key dates
| Filing date | Sep 16, 1994 |
| Grant date | Dec 23, 1997 |
| Priority date | — |
| Expiry date | Sep 16, 2014 |
Classification
- Technology area (CPC Y)Emerging Cross-Sectional Technologies
- CPC primaryY02A50/30
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Methods for stimulating gamma globin gene expression in a mammalian cell comprising contacting the cell with a compound selected from valeric acid and certain isomers, derivatives or salts thereof, including isovaleric acid, 4-pentynoic acid and methylthioacetic acid, or an inhibitor of a short chain fatty acyl CoA dehydrogenase, or an activator of protein kinase C are disclosed. The methods of the invention are particularly useful for ameliorating .beta.-globin disorders, such as sickle cell anemia or .beta.-thalassemia. The method of the invention can also be used to prevent or ameliorate malaria in a mammal. The compounds of the invention can also be used to stimulate differentiation of a cell. Pharmaceutical compositions of the active compounds of the invention are also disclosed. Screening assays for identifying agents that stimulate gamma globin gene expression in an erythroid cell, stimulate cell differentiation or stimulate transport of a short chain fatty acid, or derivative or salt thereof, into cells are also disclosed.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.