MiRNA Expression in Patients with Gaucher Disease Treated with Enzyme Replacement Therapy.

ABSTRACT: The aim of the work was to establish potential biomarkers or drug targets by analysing changes in miRNA concentration among patients with Gaucher disease (GD) compared to in healthy subjects. This study was an observational, cross-sectional analysis of 30 adult participants: 10 controls and 20 adults with GD type 1. Patients with GD type 1 were treated with enzyme replacement therapy (ERT) for at least two years. The control group was composed of healthy volunteers, unrelated to the patients, adjusted with age, sex and body mass index (BMI). The miRNA alteration between these groups was examined. After obtaining preliminary results on a group of six GD patients by the high-output method (TaqMan low-density array (TLDA)), potential miRNAs were selected for confirming the results by using th

SUBMITTER: Pawlinski L 

PROVIDER: S-EPMC7822051 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature