The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia

• Κύριοι συγγραφείς: Ekati Drakopoulou (Συγγραφέας), Eleni Papanikolaou (Συγγραφέας), Nicholas P. Anagnou (Συγγραφέας)
• Μορφή: Βιβλίο
• Έκδοση: Hindawi Limited, 2011-01-01T00:00:00Z.
• Θέματα: article
• Διαθέσιμο Online: Connect to this object online.

β-thalassemia is characterized by reduced or absence of β-globin production, resulting in anemia. Current therapies include blood transfusion combined with iron chelation. BM transplantation, although curative, is restricted by the matched donor limitation. Gene therapy, on the other hand, is promis...