Cellular and animal models for the investigation of β-thalassemia Article Swipe
Antonella Nai
,
Celia Cordero-Sánchez
,
Emanuele Tanzi
,
Alessia Pagani
,
Laura Silvestri
,
Simona Maria Di Modica
·
YOU?
·
· 2023
· Open Access
·
· DOI: https://doi.org/10.1016/j.bcmd.2023.102761
YOU?
·
· 2023
· Open Access
·
· DOI: https://doi.org/10.1016/j.bcmd.2023.102761
β-Thalassemia is a genetic form of anemia due to mutations in the β-globin gene, that leads to ineffective and extramedullary erythropoiesis, abnormal red blood cells and secondary iron-overload. The severity of the disease ranges from mild to lethal anemia based on the residual levels of globins production. Despite being a monogenic disorder, the pathophysiology of β-thalassemia is multifactorial, with different players contributing to the severity of anemia and secondary complications. As a result, the identification of effective therapeutic strategies is complex, and the treatment of patients is still suboptimal. For these reasons, several models have been developed in the last decades to provide experimental tools for the study of the disease, including erythroid cell lines, cultures of primary erythroid cells and transgenic animals. Years of research enabled the optimization of these models and led to decipher the mechanisms responsible for globins deregulation and ineffective erythropoiesis in thalassemia, to unravel the role of iron homeostasis in the disease and to identify and validate novel therapeutic targets and agents. Examples of successful outcomes of these analyses include iron restricting agents, currently tested in the clinics, several gene therapy vectors, one of which was recently approved for the treatment of most severe patients, and a promising gene editing strategy, that has been shown to be effective in a clinical trial. This review provides an overview of the available models, discusses pros and cons, and the key findings obtained from their study.
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Metadata
- Type
- review
- Language
- en
- Landing Page
- https://doi.org/10.1016/j.bcmd.2023.102761
- OA Status
- hybrid
- Cited By
- 5
- References
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- Related Works
- 10
- OpenAlex ID
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All OpenAlex metadata
Raw OpenAlex JSON
- OpenAlex ID
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https://openalex.org/W4378965599Canonical identifier for this work in OpenAlex
- DOI
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https://doi.org/10.1016/j.bcmd.2023.102761Digital Object Identifier
- Title
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Cellular and animal models for the investigation of β-thalassemiaWork title
- Type
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reviewOpenAlex work type
- Language
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enPrimary language
- Publication year
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2023Year of publication
- Publication date
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2023-05-30Full publication date if available
- Authors
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Antonella Nai, Celia Cordero-Sánchez, Emanuele Tanzi, Alessia Pagani, Laura Silvestri, Simona Maria Di ModicaList of authors in order
- Landing page
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https://doi.org/10.1016/j.bcmd.2023.102761Publisher landing page
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YesWhether a free full text is available
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hybridOpen access status per OpenAlex
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https://doi.org/10.1016/j.bcmd.2023.102761Direct OA link when available
- Concepts
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Ineffective erythropoiesis, Erythropoiesis, Thalassemia, Disease, Medicine, Anemia, Bioinformatics, Genetic enhancement, Beta thalassemia, Computational biology, Intensive care medicine, Immunology, Biology, Gene, Genetics, Internal medicineTop concepts (fields/topics) attached by OpenAlex
- Cited by
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5Total citation count in OpenAlex
- Citations by year (recent)
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2025: 2, 2024: 3Per-year citation counts (last 5 years)
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126Number of works referenced by this work
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10Other works algorithmically related by OpenAlex
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| abstract_inverted_index.β-thalassemia | 55 |
| abstract_inverted_index.erythropoiesis, | 20 |
| abstract_inverted_index.multifactorial, | 57 |
| abstract_inverted_index.pathophysiology | 53 |
| cited_by_percentile_year.max | 97 |
| cited_by_percentile_year.min | 95 |
| corresponding_author_ids | https://openalex.org/A5082550914 |
| countries_distinct_count | 1 |
| institutions_distinct_count | 6 |
| corresponding_institution_ids | https://openalex.org/I154387261, https://openalex.org/I4210133214 |
| sustainable_development_goals[0].id | https://metadata.un.org/sdg/3 |
| sustainable_development_goals[0].score | 0.6800000071525574 |
| sustainable_development_goals[0].display_name | Good health and well-being |
| citation_normalized_percentile.value | 0.84862842 |
| citation_normalized_percentile.is_in_top_1_percent | False |
| citation_normalized_percentile.is_in_top_10_percent | False |