Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies.

Clicks: 189
ID: 30668
2019
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Abstract
Beta-thalassemia and sickle cell disease (SCD) are the most prevalent monogenic diseases. These disorders are caused by quantitative or qualitative defects in the production of adult hemoglobin. Gene therapy is a potential treatment option for patients lacking an allogenic compatible hematopoietic stem cell (HSC) donor. The generation of lentiviral vectors (LVs) carrying a β-globin-like gene has revolutionized this field by allowing effective HSC transduction with no evidence of genotoxicity to date. Several clinical trials with different kinds of vector are underway worldwide; the initial results are promising with regard to sustained production of therapeutic hemoglobin, improved biological parameters, lower transfusion requirement and better quality of life. Long-term follow-up will confirm the safety of this LV-based gene therapy. Optimization of patient conditioning, HSC harvest and transduction process further improved the therapeutic potential of this approach. Novel LV-based strategies aiming at reactivating endogenous fetal hemoglobin (HbF) are also promising because elevated HbF levels can reduce the severity of both β-thalassemia and SCD. Lastly, genome-editing approaches designed to correct the disease-causing mutation or to reactivate HbF are currently under investigation. Here, we discuss the clinical outcomes of current LV-based gene addition trials and the potential advantages of novel alternative therapeutic strategies.
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magrin2019lentiviralblood Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Magrin, Elisa;Miccio, Annarita;Cavazzana, Marina;
Journal Blood
Year 2019
DOI
blood.2019000949
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