Grant 24


Research Institution: ​Valentia BioPharma, Spain

Principle Investigator:

​Doctor López Caste

Type: ​International

Project title: ​Generation of Drosophila-Based Biomedical Models of FSHD

Status: Completed

Summary

This project was directed at developing a rapid, miniature system in fruit flies, for screening of potential drugs for treatment of humans with FSHD. Their first attempts to achieve fruit fly based FSHD models were focussed on the expression of the full-length toxic DUX4 protein. However, the full length-DUX4 protein expression showed toxic effects early in the fly development. Their work is now focussing on expression of shorter forms of DUX4 protein, in order to avoid its high toxicity, but retaining DUX4 protein disease linked-effects for further effective drug testing.