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Genome Editing for β-Hemoglobinopathies: Advances and Challenges
β-hemoglobinopathies are the most common genetic disorders worldwide and are caused by mutations affecting the production or the structure of adult hemoglobin. Patients affected by these diseases suffer from anemia, impaired oxygen delivery to tissues, and multi-organ damage. In the absence of a com...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2021
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7865242/ https://www.ncbi.nlm.nih.gov/pubmed/33525591 http://dx.doi.org/10.3390/jcm10030482 |