Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8648043 | Blood Cells, Molecules, and Diseases | 2018 | 12 Pages |
Abstract
Nonetheless, genetic studies have been successful in characterizing the key variants and pathways involved in HbF regulation, providing new therapeutic targets for HbF reactivation. BCL11A has been established as a quantitative repressor, and progress has been made in manipulating its expression using genomic and gene-editing approaches for therapeutic benefits. Recent discoveries and understanding in the mechanisms associated with ineffective and abnormal erythropoiesis have also provided additional therapeutic targets, a couple of which are currently being tested in clinical trials.
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Authors
Swee Lay Thein,