Emerging Therapies

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dc.contributor.authorGuerra, Amaliris
dc.contributor.authorMusallam, Khaled M.
dc.contributor.authorTaher, Ali T.
dc.contributor.authorRivella, S. B.
dc.contributor.departmentInternal Medicine
dc.contributor.facultyFaculty of Medicine (FM)
dc.contributor.institutionAmerican University of Beirut
dc.date.accessioned2025-01-24T11:52:25Z
dc.date.available2025-01-24T11:52:25Z
dc.date.issued2018
dc.description.abstractAt 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 approaches for the treatment of hemoglobinopathies caused by disruptions of β-globin. © 2017 Elsevier Inc.
dc.identifier.doihttps://doi.org/10.1016/j.hoc.2017.11.002
dc.identifier.eid2-s2.0-85039414165
dc.identifier.pmid29458736
dc.identifier.urihttp://hdl.handle.net/10938/31058
dc.language.isoen
dc.publisherW.B. Saunders
dc.relation.ispartofHematology/Oncology Clinics of North America
dc.sourceScopus
dc.subjectGene transfer
dc.subjectHemichromes
dc.subjectNew therapies
dc.subjectTrap ligands
dc.subjectΒ-globin
dc.subjectΒ-thalassemia
dc.subjectAnimals
dc.subjectBeta-globins
dc.subjectBeta-thalassemia
dc.subjectClinical trials as topic
dc.subjectCombined modality therapy
dc.subjectDisease management
dc.subjectDrug discovery
dc.subjectDrug evaluation, preclinical
dc.subjectErythropoiesis
dc.subjectHumans
dc.subjectBeta globulin
dc.subjectHemoglobin beta chain
dc.subjectAutologous bone marrow transplantation
dc.subjectBeta thalassemia
dc.subjectDrug development
dc.subjectExperimental therapy
dc.subjectGene therapy
dc.subjectHematopoietic stem cell transplantation
dc.subjectHuman
dc.subjectNonhuman
dc.subjectPreclinical study
dc.subjectPriority journal
dc.subjectRandomized controlled trial (topic)
dc.subjectReview
dc.subjectAnimal
dc.subjectBlood
dc.subjectClinical trial (topic)
dc.subjectGenetics
dc.subjectMultimodality cancer therapy
dc.subjectProcedures
dc.titleEmerging Therapies
dc.typeReview

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