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Curing Hemoglobinopathies: Challenges and Advances of Conventional and New Gene Therapy Approaches
Inherited hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell disease (SCD), are the most common monogenic diseases worldwide, with a global carrier frequency of over 5%.1 With migration, they are becoming more common worldwide, making their management and care an increasing concer...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Università Cattolica del Sacro Cuore
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6827604/ https://www.ncbi.nlm.nih.gov/pubmed/31700592 http://dx.doi.org/10.4084/MJHID.2019.067 |