Article

The therapeutic potential of genome editing for β-thalassemia

2015-12-11

Abstract excerpt

The rapid advances in the field of genome editing using targeted endonucleases have called considerable attention to the potential of this technology for human gene therapy. Targeted correction of disease-causing mutations could ensure lifelong, tissue-specific expression of the relevant gene, thereby alleviating or resolving a specific disease phenotype. In this review, we aim to explore the potential of this tec...

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Literature Corpus work: 5b611e08-303d-592e-a273-82aeb8dabaae
DOI: 10.12688/f1000research.7087.1

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