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Trikafta, a CFTR modulator, is increasingly used to treat children with cystic fibrosis (CF), but its effects remain unclear. This study evaluated the cellular mechanisms of Trikafta in young children (< 11 years) with CF using a multi-omic approach. Seventeen children with CF (median age 8.4 years,...
ORGANISM(S): Homo sapiens (Human) 
2026-08-03 | PXD060637 | Pride
Early, low risk IPSS (International Prognostic Scoring System) myelodysplasia (MDS) is a heterogeneous disorder where the molecular and cellular haematopoietic defects are poorly understood. To gain insight into this condition, we analyzed gene expression profiles of marrow CD34+ progenitor cells fr...
ORGANISM(S): Homo sapiens 
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