A systematic review of clinical trials for gene therapies for β-hemoglobinopathy around the world

Rós, Felipe Augusto; Couto, Samuel Campanelli Freitas; Milhomens, Jonathan; Ovider, Ian; Maio, Karina Tozatto; Jennifer, Viviane; Ramos, Rodrigo Nalio; Picanço-Castro, Virginia; Kashima, Simone; Calado, Rodrigo T.; Barros, Luciana Rodrigues Carvalho; Rocha, Vanderson

doi:10.1016/j.jcyt.2023.05.006

Cytotherapy

2023

DOI: 10.1016/j.jcyt.2023.05.006

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A systematic review of clinical trials for gene therapies for β-hemoglobinopathy around the world

Felipe Augusto Rós,

Samuel Campanelli Freitas Couto,

Jonathan Milhomens

et al.

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2024

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Cited by 3 publications

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Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Locatelli,

Lang,

Wall

et al. 2024

N Engl J Med

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Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Locatelli,

Lang,

Wall

et al. 2024

N Engl J Med

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Genome Editing Therapy for the Blood: Ex Vivo Success and In Vivo Prospects

George,

Sahu,

de Oñate

et al. 2024

The CRISPR Journal

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β-Thalassemia in Bangladesh: Current Status and Future Perspectives

Mitro,

Hossain,

Rahman

et al. 2024

Thalassemia Reports

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β-thalassemia, a life-threatening inheritable hemoglobin disorder caused by mutations in the HBB gene, poses a significant public health challenge in the world. Although no comprehensive work has been carried out in Bangladesh, the world prevalence and small-scale works indicated the possibility of a high prevalence of this disease in the country. Therefore, this review aims to explore the present situation of β-thalassemia in Bangladesh and propose approaches to mitigate its impact in the future. Limited awareness, a high incidence of consanguineous marriage, and inadequate access to healthcare are possible factors responsible for the high prevalence of thalassemia in Bangladesh, while the absence of public health policy and a national health insurance system further exacerbate the situation. The understanding of the genetic landscape and modern treatment strategies for β-thalassemia is hindered by the lack of comprehensive data on the mutation spectrum. In addition to conventional therapy such as blood transfusion, advanced practices such as splenectomy, hematopoietic stem cell transplantation, and emerging therapies such as gene therapy show promise for future cures but have yet to be widely implemented in this country. To effectively address the challenges of β-thalassemia, it is crucial to adopt comprehensive strategies, including a public awareness campaign, public health intervention, mandatory premarital screening, genetic counselling, and a national thalassemia prevention program. Additionally, understanding the spectrum of mutations and new therapeutic interventions is crucial for advanced healthcare strategies.

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A systematic review of clinical trials for gene therapies for β-hemoglobinopathy around the world

Cited by 3 publications

References 38 publications

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Genome Editing Therapy for the Blood: Ex Vivo Success and In Vivo Prospects

β-Thalassemia in Bangladesh: Current Status and Future Perspectives

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