Beta-thalassemia future or investigational therapies: Difference between revisions
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{{Beta-thalassemia}} | {{Beta-thalassemia}} | ||
{{CMG}}; {{AE}} {{MHP}} | |||
==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<ref name="pmid30700149">{{cite journal |vauthors=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 |title=Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation |journal=Hum Gene Ther |volume=30 |issue=6 |pages=753–761 |date=June 2019 |pmid=30700149 |doi=10.1089/hum.2018.178 |url=}}</ref>. | |||
==References== | ==References== | ||
{{reflist|2}} | {{reflist|2}} | ||
Latest revision as of 05:58, 25 August 2023
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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.