| DOI | Resolve DOI: https://doi.org/10.1182/blood-2024-211315 |
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| Author | Search for: Ketharnathan, Sarada; Search for: Dogan, Serkan; Search for: Pokharel, Sujata; Search for: Prykhozhij, Sergey V; Search for: Cordeiro, Anna; Search for: Alecu, Irina; Search for: Mannherz, William; Search for: Liebman, Mira; Search for: Leung, Elaine W.; Search for: Bennett, SteffanyORCID identifier: https://orcid.org/0000-0001-7944-5800; Search for: Cuperlovic-Culf, Miroslava1ORCID identifier: https://orcid.org/0000-0002-9483-8159; Search for: Agarwal, Suneet; Search for: Dror, Yigal; Search for: Berman, Jason N. |
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| Affiliation | - National Research Council Canada. Digital Technologies
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| Format | Text, Article |
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| Abstract | Inherited bone marrow failure syndromes (IBMFS) are a clinically diverse group of rare genetic disorders characterized by cytopenia of one or more hematopoietic lineages. IBMFS account for 10-15% of marrow aplasia and >50% of chronic pediatric bone marrow failure disorders. Definitive treatment for all IBMFS currently requires allogeneic hematopoietic stem cell transplantation. However, post-transplant complications such as organ toxicity and engraftment failure are common in this population. A thorough understanding of the underlying disease biology would enable the development of targeted therapeutic interventions that could rescue marrow failure, and potentially prevent progression to myeloid malignancies. We hypothesized that in addition to the transcriptional dysregulation, known to underpin IBMFS, perturbations in metabolism are essential for the transition of hematopoietic cells from a state of hypo-proliferation in IBMFS to one of hyperproliferation in leukemia. Given the rarity of IBMFS, large numbers of primary human samples are not readily available for mechanistic studies, warranting the use of animal models. Zebrafish (Danio rerio) are ideal given their highly conserved and rapid hematopoiesis and access to early blood progenitors. |
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| Publication date | 2024-11-05 |
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| Publisher | American Society of Hematology |
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| In | |
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| Language | English |
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| Peer reviewed | Yes |
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| Export citation | Export as RIS |
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| Report a correction | Report a correction (opens in a new tab) |
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| Record identifier | fea7e904-1d6c-495f-8995-a77c188831d7 |
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| Record created | 2024-12-12 |
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| Record modified | 2024-12-12 |
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