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Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease

15 Jun 2016

Abstract excerpt

Recent advances in gene therapy and genome-engineering technologies offer the opportunity to correct sickle cell disease (SCD), a heritable disorder caused by a point mutation in the β-globin gene. The developmental switch from fetal γ-globin to adult β-globin is governed in part by the transcription factor (TF) BCL11A. This TF has been proposed as a therapeutic target for reactivation of γ-globin and concomitant...

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PMID: 27299299
DOI: 10.1002/ajh.24449

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