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Gene therapy for hemoglobinopathies: progress and challenges

Hemoglobinopathies are genetic inherited conditions that originate from the lack or malfunction of the hemoglobin (Hb) protein. Sickle cell disease (SCD) and thalassemia are the most common forms of these conditions. The severe anemia combined with complications that arise in the most affected patie...

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Bibliografiska uppgifter
Huvudupphovsmän: Dong, Alisa, Rivella, Stefano, Breda, Laura
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2013
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC3716457/
https://ncbi.nlm.nih.gov/pubmed/23337292
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.trsl.2012.12.011
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