β-Globin Sleeping Beauty Transposon Reduces Red Blood Cell Sickling in a Patient-Derived CD34(+)-Based In Vitro Model

The ultimate goal of gene therapy for sickle cell anemia (SCA) is an improved phenotype for the patient. In this study, we utilized bone marrow from a sickle cell patient as a model of disease in an in vitro setting for the hyperactive Sleeping Beauty transposon gene therapy system. We demonstrated...

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Detalles Bibliográficos
Autores principales: Sjeklocha, Lucas M., Wong, Phillip Y.-P., Belcher, John D., Vercellotti, Gregory M., Steer, Clifford J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2013
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3832362/
https://www.ncbi.nlm.nih.gov/pubmed/24260386
http://dx.doi.org/10.1371/journal.pone.0080403

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3832362/
https://www.ncbi.nlm.nih.gov/pubmed/24260386
http://dx.doi.org/10.1371/journal.pone.0080403

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