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The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges
Hemoglobinopathies, including β-thalassemia and sickle cell disease (SCD), are a heterogeneous group of commonly inherited disorders affecting the function or levels of hemoglobin. Disease phenotype can be severe with substantial morbidity and mortality. Bone marrow transplantation is curative, but...
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| Publicado no: | Ther Adv Hematol |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
SAGE Publications
2016
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5026290/ https://ncbi.nlm.nih.gov/pubmed/27695619 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/2040620716653729 |
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