Prioritization of novel ADPKD drug candidates from disease-stage specific gene expression profiles.

ABSTRACT:

Background

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is one of the most common causes of end-stage renal failure, caused by mutations in PKD1 or PKD2 genes. Tolvaptan, the only drug approved for ADPKD treatment, results in serious side-effects, warranting the need for novel drugs.

Methods

In this study, we applied RNA-sequencing of Pkd1cko mice at different disease stages, and with/without drug treatment to identify genes involved in ADPKD progression that were further used to identify novel drug candidates for ADPKD. We followed an integrative computational approach using a combination of gene expression profiling, bioinformatics and cheminformatics data.

Findings

We identified 1162 genes that had a normalized expression after treating the mice with drugs

SUBMITTER: Malas TB 

PROVIDER: S-EPMC7000333 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature