The therapeutic potential of genome editing for β-thalassemia [version 1; referees: 2 approved]
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, there...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
F1000 Research Ltd
2015-12-01
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| Series: | F1000Research |
| Subjects: | |
| Online Access: | http://f1000research.com/articles/4-1431/v1 |