2017, new articles
ASH meeting; see our papers in the event section
Growth delay is common in children with chronic kidney disease (CKD), often associated with poor quality of life. Our study suggests that hepcidin appears to be related to pathways of growth retardation in CKD and that investigation of hepcidin lowering therapies in juvenile CKD is warranted.
A new powerful tool generated by the Blobel lab (see articles Deng W et al, Cell 2012 and Deng W et al, Cell 2014) is able to increase fetal hemoglobin in cells from sickle cell anemia patient in vitro.
Our research suggests that synthetic peptides called minihepcidins may potentially treat two serious genetic blood diseases in children and adults. Although those diseases, beta-thalassemia and polycythemia vera, have opposite effects on red blood cell production, treating animals with minihepcidin helps to restore normal levels of red blood cells and reduces spleen enlargement.
Our study shows that an antisense oligonucleotide targeting Tmprss6 combined with the oral iron chelator DFP, is more powerful in reducing hepatic iron stores than either therapy alone, independently from dietary iron content.
Sultan bin Khalifa International Thalassemia Award - Innovative Medical Research award to Dr. Stefano Rivella