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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...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Mary Ann Liebert, Inc., publishers
2018
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6196755/ https://www.ncbi.nlm.nih.gov/pubmed/30200783 http://dx.doi.org/10.1089/hum.2018.122 |