Health Dictionary
Reliable, easy-to-read patient information on common medical conditions — written in plain language, free of jargon, and grounded in current evidence. Browse the most frequent diagnoses, understand what they mean for everyday life, and learn which steps and questions help you have a focused conversation with your doctor.
Beta-thalassemia future or investigational therapies
Introduction
Overview
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
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
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.
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Maryam Hadipour, M.D.[2]