Posted by Friends of FSH Research on Apr 2, 2019
The following five grants have been approved so far this calendar year, totaling $350,000. This is only made possible by your continued generous support of research over the years, and our outstanding Scientific Advisory Board members' dedication to focus on finding a cure.
Travel Reimbursement for Clinical Study Participants
Michael Kyba PhD
These funds will be drawn upon to reimburse individuals for who need to travel to participate in our clinical studies on FSHD, as well as to provide a small remuneration in view of incidental costs incurred by all participants. Studies: Metabolic Rate and Exercise Tolerance in Adults with Facioscapulohumeral Muscular Dystrophy (Manda Keller-Ross, PI) and Tissue Biopsies for the Study of FSHD (Michael Kyba, PI).
In vivo model to validate drugs targeting DUX4 enhancer/promoter activity
Yosuke Hiramuki PhD
This study complements current drug validation studies, providing important insight to developing FSHD therapeutics in the living organism, and serve the field as a mouse model platform that can be utilized for new drug development.
DUX4 mRNA silencing using CRISPR-Cas13
Afrooz Rashnonejad PhD
Development of an alternative CRISPR system – based on the Cas13 enzyme – that can be targeted to the DUX4 mRNA instead of DNA.
Base editing of DUX4 somatic polyadenylation signal – towards new therapeutics for FSHD
Darina Šikrová MSc
Modification of the STOP signal of the DUX4 gene in a way that it cannot be longer recognized by the reading cellular machinery, which should lead to inefficient production of DUX4 and ultimately its lower levels in skeletal muscle cells.
Young Investigator Travel Grant
Friends is pleased to help support FSHD investigators by providing funds for travel to the 2019 FSH Society International Research Congress. Afrooz Rashnonejad (a newly funded investigator mentioned above) is one recipient of a travel grant from these funds. This year's meeting will take place June 19-20, 2019 at Pharo Palace, Marseille, France.
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