Emerging Therapies.
Ontology highlight
ABSTRACT:
SUBMITTER: Guerra A
PROVIDER: S-EPMC5823282 | biostudies-literature | 2018 Apr
REPOSITORIES: biostudies-literature
Publications
Emerging Therapies.
Guerra Amaliris A Musallam Khaled M KM Taher Ali T AT Rivella Stefano S
Hematology/oncology clinics of North America 20171215 2
At present, the only definitive cure for β-thalassemia is a bone marrow transplant (BMT); however, HLA-blood-matched donors are scarcely available. Current therapies undergoing clinical investigation with most potential for therapeutic benefit are the β-globin gene transfer of patient-specific hematopoietic stem cells followed by autologous BMT. Other emerging therapies deliver exogenous regulators of several key modulators of erythropoiesis or iron homeostasis. This review focuses on current ap ...[more]
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