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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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| Publicat a: | Blood |
|---|---|
| Autors principals: | , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Society of Hematology
2016
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| Matèries: | |
| Accés en línia: | 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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