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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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Detalles Bibliográficos
Publicado en:Ther Adv Hematol
Main Authors: Goodman, Michael A., Malik, Punam
Formato: Artigo
Idioma:Inglês
Publicado: SAGE Publications 2016
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Acceso en liña: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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