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Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects
The beta-thalassemias are inherited anemias caused by mutations that severely reduce or abolish expression of the beta-globin gene. Like sickle cell disease, a related beta-globin gene disorder, they are ideal candidates for performing a genetic correction in patient hematopoietic stem cells (HSCs)....
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| Publicado en: | Hum Gene Ther |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
Mary Ann Liebert, Inc.
2016
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4994056/ https://ncbi.nlm.nih.gov/pubmed/27021486 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2016.037 |
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