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Gene Therapy for Hemoglobinopathies
Gene therapy for β-thalassemia and sickle-cell disease is based on transplantation of genetically corrected, autologous hematopoietic stem cells. Preclinical and clinical studies have shown the safety and efficacy of this therapeutic approach, currently based on lentiviral vectors to transfer a β-gl...
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| Publicado no: | Hum Gene Ther |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Mary Ann Liebert, Inc., publishers
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6196755/ https://ncbi.nlm.nih.gov/pubmed/30200783 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2018.122 |
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