| 摘要 |
<p>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 β-globin disorders, such as sickle cell anemia or β-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 acive 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.</p> |
