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Gene Therapy and Gene Editing for β-Thalassemia. | LitMetric

Gene Therapy and Gene Editing for β-Thalassemia.

Hematol Oncol Clin North Am

Department of Hematology, St. Jude Children's Research Hospital, 262 Danny Thomas Place, MS #355, Memphis, TN 38105, USA. Electronic address:

Published: April 2023

After many years of intensive research, emerging data from clinical trials indicate that gene therapy for transfusion-dependent β-thalassemia is now possible. Strategies for therapeutic manipulation of patient hematopoietic stem cells include lentiviral transduction of a functional erythroid-expressed β-globin gene and genome editing to activate fetal hemoglobin production in patient red blood cells. Gene therapy for β-thalassemia and other blood disorders will invariably improve as experience accumulates over time. The best overall approaches are not known and perhaps not yet established. Gene therapy comes at a high cost, and collaboration between multiple stakeholders is required to ensure that these new medicines are administered equitably.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10355137PMC
http://dx.doi.org/10.1016/j.hoc.2022.12.012DOI Listing

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