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The therapeutic potential of genome editing for β-thalassemia
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...
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| Publicado no: | F1000Res |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
F1000Research
2015
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4753996/ https://ncbi.nlm.nih.gov/pubmed/26918126 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.12688/f1000research.7087.1 |
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