Beta-thalassemia future or investigational therapies
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Beta-thalassemia Microchapters |
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Maryam Hadipour, M.D.[2]
Overview
Promising future therapies for beta-thalassemia major include gene therapy, stem cell transplantation, and novel approaches targeting gene editing or gene regulation to enhance the production of functional beta-globin chains.
Future or investigational therapies
Promising future therapies for beta-thalassemia major include:
- Gene therapy
- Stem cell transplantation
- Novel approaches targeting gene editing or gene regulation to enhance the production of functional beta-globin chains[1].
References
- ↑ Coquerelle S, Ghardallou M, Rais S, Taupin P, Touzot F, Boquet L, Blanche S, Benaouadi S, Brice T, Tuchmann-Durand C, Ribeil JA, Magrin E, Lissillour E, Rochaix L, Cavazzana M, Durand-Zaleski I (June 2019). "Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation". Hum Gene Ther. 30 (6): 753–761. doi:10.1089/hum.2018.178. PMID 30700149.