Novel Inducers of Fetal Globin Identified through High Throughput Screening (HTS) Are Active In Vivo in Anemic Baboons and Transgenic Mice

High-level fetal (γ) globin expression ameliorates clinical severity of the beta (β) hemoglobinopathies, and safe, orally-bioavailable γ-globin inducing agents would benefit many patients. We adapted a LCR-γ-globin promoter-GFP reporter assay to a high-throughput robotic system to evaluate five dive...

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Detalhes bibliográficos
Publicado no:PLoS One
Main Authors: Boosalis, Michael S., Sangerman, Jose I., White, Gary L., Wolf, Roman F., Shen, Ling, Dai, Yan, White, Emily, Makala, Levi H., Li, Biaoru, Pace, Betty S., Nouraie, Mehdi, Faller, Douglas V., Perrine, Susan P.
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2015
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4694699/
https://ncbi.nlm.nih.gov/pubmed/26713848
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0144660
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