Article

Clinical-Scale Genome Editing of the Human BCL11A Erythroid Enhancer for Treatment of the Hemoglobinopathies

2015-12-03

Abstract excerpt

Abstract We describe here a fundamentally novel way to develop a disease therapeutic: combining genome-wide association studies (GWAS) with targeted genome editing to create, in a clinically compliant setting, a disease-ameliorating genotype in the patient's own cells. In β-thalassemia, elevated levels of fetal hemoglobin (HbF) lessen or eliminate disease symptoms, thus making a reversal of HbF silencing in patien...

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Literature Corpus work: d660edf0-0a5a-516e-b85d-4e662c908340
DOI: 10.1182/blood.v126.23.204.204

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Clinical-Scale Genome Editing of the Human BCL11A Erythroid Enhancer for Treatment of the HemoglobinopathiesDOI 10.1182/blood.v126.23.204.204Read

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