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FARAFARA Cure FA

UAB Researchers Receive Funding to Seek Biomarkers in Friedreich's Ataxia

The University of Alabama at Birmingham researchers have received funding to seek and validate biomarkers for Friedreich's Ataxia with a two-year, $140,000-a-year grant funded by The Friedreich's Ataxia Research Alliance (FARA) and FARA Ireland.

Discovery of validated biomarkers is a high priority because they are invaluable to help speed the development and clinical evaluation of potential drugs. FARA's treatment pipeline already includes nine potential drugs or treatments that are in human clinical trials, seven more that are in preclinical testing, and four others in the early discovery phase.

Marek Napierala, Ph.D., assistant professor in the UAB Department of Biochemistry and Molecular Genetics, UAB Stem Cell Institute, and Jill Butler, Ph.D., instructor in Biochemistry and Molecular Genetics, are co-investigators. ‪

In preliminary work for the grant application, Butler and Napierala looked at RNA gene expression in patient and control fibroblast cells. They identified several gene expression variants as candidate biomarkers, and they now are working to validate those candidates in neuronal and cardiac Friedreich’s ataxia cell line models and to adapt these biomarkers for routine testing in a clinical setting. They measure protein expression levels using reverse phase protein arrays, and they have future plans to look for changes in microRNAs in the Friedreich’s ataxia cells. MicroRNAs are short sequences of RNA that act as robust agents of gene regulation in cells.

“For this research to be successful and make it to the clinic,” Napierala said, “the test or measurement needs to be easily done.” Ideal biomarkers also have to be safe, cost effective and easily adapted from research laboratory testing to the clinic.

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The FARA Ambassador Program

The FARA Ambassador Program

The FARA Ambassadors are a united team of patient volunteers living with FA who are committed to supporting FARA in the search for a treatment and cure.
 
Together we seek to know more about FA, and FARA so we can be prepared to represent the community when the opportunity arises; speaking at events, to volunteers, potential donors, scientific groups, pharma partners, media interviews and other awareness and fundraising opportunities. We believe support is key to continued success toward our ultimate goal of treatment and a cure. Participants in the FARA Ambassador Program are passionate about building and upholding relationships within the FA community.
 
The FARA Ambassadors are positive, supportive, peer representatives for the FA community, actively raising awareness and funds for FARA. To learn more about the FARA Ambassador Program or to have a FARA Ambassador speak at your event, please contact: info@cureFA.org.

 

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