ArticleBritish journal of haematology2014
L-Leucine improves the anaemia in models of Diamond Blackfan anaemia and the 5q- syndrome in a TP53-independent way.
Article in British journal of haematology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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Who cites it
21 citing papers in PubMed, 31 citations in OpenAlex.
- Established and emerging non-cellular therapies in inherited bone marrow failure syndromes.Frontiers in immunology · 2026Review
- Review
- The transcriptomic landscape of normal and ineffective erythropoiesis at single-cell resolution.Blood advances · 2023Article
- Article
- Branched-chain amino acids are linked with iron metabolism.Annals of translational medicine · 2020Article
- Diamond-Blackfan anemia.Blood · 2020Review
- Loss ofG3 (Bethesda, Md.) · 2019Article
- Modeling hematopoietic disorders in zebrafish.Disease models & mechanisms · 2019Review
- Cancer Biogenesis in Ribosomopathies.Cells · 2019Review
- How Ribosomes Translate Cancer.Cancer discovery · 2017Review
- Modeling Myeloid Malignancies Using Zebrafish.Frontiers in oncology · 2017Review
- Understanding the regulation of vertebrate hematopoiesis and blood disorders - big lessons from a small fish.FEBS letters · 2016Review
- Probing the mechanisms underlying human diseases in making ribosomes.Biochemical Society transactions · 2016Review
- RPL27A is a target of miR-595 and may contribute to the myelodysplastic phenotype through ribosomal dysgenesis.Oncotarget · 2016Article
- The Ribosome Biogenesis Protein Nol9 Is Essential for Definitive Hematopoiesis and Pancreas Morphogenesis in Zebrafish.PLoS genetics · 2015Article
- The mTORC1/4E-BP pathway coordinates hemoglobin production with L-leucine availability.Science signaling · 2015Article
- L-leucine partially rescues translational and developmental defects associated with zebrafish models of Cornelia de Lange syndrome.Human molecular genetics · 2015Article
- Whole-genome RNAi screen highlights components of the endoplasmic reticulum/Golgi as a source of resistance to immunotoxin-mediated cytotoxicity.Proceedings of the National Academy of Sciences of the United States of America · 2015Article
- Article
- Ribosomopathies: Global process, tissue specific defects.Rare diseases (Austin, Tex.) · 2015Review
Corrections and comments
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Authors and funding
9 authors at 4 institutions in 2 countries.
Funding
Abstract
Haploinsufficiency of ribosomal proteins (RPs) and upregulation of the tumour suppressor TP53 have been shown to be the common basis for the anaemia observed in Diamond Blackfan anaemia and 5q- myelodysplastic syndrome. We previously demonstrated that treatment with L-Leucine resulted in a marked improvement in anaemia in disease models. To determine if the L-Leucine effect was Tp53-dependent, we used antisense MOs to rps19 and rps14 in zebrafish; expression of tp53 and its downstream target cdkn1a remained elevated following L-leucine treatment. We confirmed this observation in human CD34+ cells. L-Leucine thus alleviates anaemia in RP-deficient cells in a TP53-independent manner.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.