Novel therapeutic candidates, identified by molecular modeling, induce γ-globin gene expression in vivo()

The β-hemoglobinopathies and thalassemias are serious genetic blood disorders affecting the β-globin chain of hemoglobin A (α(2)β(A)(2)). Their clinical severity can be reduced by enhancing expression of fetal hemoglobin (γ-globin), producing HbF (α(2)γ(2,)). In studies reported here, γ-globin induc...

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Main Authors: Boosalis, Michael S., Castaneda, Serguei A., Trudel, Marie, Mabaera, Rodwell, White, Gary L., Lowrey, Christopher H., Emery, David W., Mpollo, Marthe-Sandrine Eiymo Mwa, Shen, Ling, Wargin, William A., Bohacek, Regine, Faller, Douglas V., Perrine, Susan P.
Format: Artigo
Sprog:Inglês
Udgivet: 2011
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3148318/
https://ncbi.nlm.nih.gov/pubmed/21641240
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bcmd.2011.04.008
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