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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...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
F1000Research
2015
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4753996/ https://www.ncbi.nlm.nih.gov/pubmed/26918126 http://dx.doi.org/10.12688/f1000research.7087.1 |