Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for Β-Hemoglobinopathies
CELL STEM CELL(2021)
摘要
Sickle cell disease (SCD) is caused by a well-defined point mutation in the β-globin gene and therefore is an optimal target for hematopoietic stem cell (HSC) gene-addition/editing therapy. In HSC gene-addition therapy, a therapeutic β-globin gene is integrated into patient HSCs via lentiviral transduction, resulting in long-term phenotypic correction. State-of-the-art gene-editing technology has made it possible to repair the β-globin mutation in patient HSCs or target genetic loci associated with reactivation of endogenous γ-globin expression. With both approaches showing signs of therapeutic efficacy in patients, we discuss current genetic treatments, challenges, and technical advances in this field.
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关键词
hematopoietic stem cells,lentiviral vector,gene therapy,genome editing,CRISPR-Cas9,sickle cell disease
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