Featured Scientists

Name: Elisabetta (Liz) Soragni

Where do you work? I work for FARA. I am the new Associate Director of Research and stated my position in October of last year.

How long have you been working on FA and who was the first fellow FA researcher you met? I have been working on FA for about 14 years. The first FA researcher I met was Dr. Joel Gottesfeld. I interviewed for a position in his lab at The Scripps Research Institute in La Jolla the year after his lab started working on FA.

Name: Marcondes C. França Jr, MD, PhD

Where do you work? Department of Neurology, University of Campinas (UNICAMP), Brazil

How long have you been working on FA and who was the first fellow FA researcher you met? I have been working with FA for more than ten years. The initial contact I had with FA was during my fellowship in neuromuscular disorders. During my Ph.D. (2005-2008) under the supervision of Prof Iscia Lopes- Cendes, I kept working in the field.

What got you interested in FA research? FA deeply impacts the quality of life of affected patients, most of them as kids, teenagers, or young adults. In my personal view, the only way to move forward and find effective treatments is by doing research. This is my real motivation – try to help these individuals.

Dr. Ben Deverman

By Matt LaFleur

“Please, just Ben.”

The first thing I asked Dr. Ben Deverman is whether he preferred to be called “Dr. Ben” or “Dr. Deverman.” He seemed relieved as he gave me his answer, and I knew that my job would be easy: I am lucky to introduce him.

One of the most exciting fields of scientific research is gene therapy, a term that leaves most of us suffering with FA feeling awe and wonder. If FA is, by definition, a genetic anomaly, then the science of correcting genes seems extremely promising for FA.

The exciting promise of gene therapy was delivered to us at the FA symposium by Ben Deverman, PhD, Senior Research Scientist, director of the CLOVER (CLARITY, Optogenetics and Vector Engineering Research) Center within the Beckman Institute at Caltech in Pasadena, CA:

Name: James (Jim) Rusche

Where do you work? I am retired from directing Research and Development for a Biotech company that was active in FA drug development, Repligen Corporation. They are no longer active in therapeutic development projects.

How long have you been working on FA and who was the first fellow FA researcher you met? I started working on FA in 2007 when I was made aware of the research findings of Dr. Joel Gottesfeld at Scripps Research Institute. We started a collaborative research and development program to advance an approach Dr. Gottesfeld had identified, which involved increasing frataxin expression.

What got you interested in FA research? As a leader in a commercial company, I was charged with finding opportunities to advance new therapies for rare diseases in which there was no current treatment. Rare diseases with recently identified single gene defects like FA were important opportunities to develop new treatments and help patients.