SCREENING TARGETS AND COMPOSITIONS AND METHODS FOR TREATMENT OF CILIOPATHY DISORDERS

摘要

The present disclosure provides methods of screening and compositions and methods for treating ciliopathy diseases. Methods are provided for screening for molecules to treat ciliopathy disorders by measuring the activity of ubiquitin-proteasome system (UPS)-mediated protein degradation in the presence and the absence of a candidate molecule, wherein an increase of the activity in the presence of the candidate molecule identifies the molecule as a potential therapeutic. The methods provided also include measuring the activity of a ubiquitin peptidase or a Zic family member 1 (ZIC1) gene product, in the presence and absence of a candidate molecule, wherein a decrease in activity in the presence of the molecule identifies it as a potential therapeutic.

主权项

1. A method of screening for a therapeutic molecule to treat a ciliopathy disorder, the method comprising:
measuring the activity of a ubiquitin-proteasome system (UPS)-mediated protein degradation in the presence and the absence of a candidate molecule, wherein an increase of the UPS-mediated protein degradation activity in the presence of the candidate molecule identifies the candidate molecule as a potential therapeutic molecule to treat a ciliopathy disorder.