Citation:
Brit J Haematol. 2014;[Epub ahead of print] doi:10.1111/bjh.13069
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.
Epub:
Yes
Link to Publication:
http://onlinelibrary.wiley.com/doi/10.1111/bjh.13069/abstract
Organism or Cell Type:
zebrafish