A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction

A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction

Gene therapy might fall short in achieving a complete reversion of the β-thalassemic phenotype due to current limitations in vector design and myeloablative regimen. Following gene transfer, all or a large proportion of erythroid cells might express suboptimal levels of β-globin, impairing the thera...

Full description

Bibliographic Details
Main Authors: Zuccato, Cristina, Breda, Laura, Salvatori, Francesca, Breveglieri, Giulia, Gardenghi, Sara, Bianchi, Nicoletta, Brognara, Eleonora, Lampronti, Ilaria, Borgatti, Monica, Rivella, Stefano, Gambari, Roberto
Format: Online
Language:English
Published: Springer-Verlag 2012
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3389244/

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3389244/

Similar Items