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Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease
Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent and severe monogenic disorder, SCD has been long considered a logical candidate for gene therapy. Significant progress has been made in moving toward this goal. These efforts have provided substantial insight...
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| Publicado no: | Blood |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Hematology
2016
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4760089/ https://ncbi.nlm.nih.gov/pubmed/26758916 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2015-09-618587 |
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