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Mitochondrial Disease Research Inspires Helios Scholar at TGen

Abby Moskowitz investigated gene therapy as a treatment option for Mito and won first place at the Helios Scholars at TGen Symposium for her oral presentation.

This summer, Abby Moskowitz, a senior biochemistry and global health major at Arizona State University, investigated gene therapy as a treatment option for Mitochondrial Disease research through her Helios Scholars at TGen internship, and her work and inspiration will continue long after the summer is over.

"Interning at the Center for Rare Childhood Disorders at TGen has been one of the most rewarding and eye-opening experiences," Abby explained. "I learned the true meaning of personalized medicine and the positive impact it has on a child and their family."

Working under the mentorship of Research Assistant Professor Isabelle Schrauwen, Abby compared two unrelated families that both have a mutation associated with Mitochondrial Disease to determine if antisense oligotherapy would be an effective treatment plan. Antisense oligotherapy targets a specific place in the genome known to cause a disease, effectively turning the gene "off."

"Abby's summer project was part of a larger project aimed to improve mitochondrial function in certain patients with Mitochondrial Disease, caused by mutations in the MTFMT gene," Dr. Schrauwen said. "I think our research could open up a path towards treatment, and give hope for the future for many patients."

Abby was one of seven Helios Scholars selected to be a speaker at the Helios Scholars Symposium on July 24, and she was awarded first place for her oral presentation. Abby will be continuing her work on Mitochondrial Disease through the fall as a volunteer intern.

"Abby was always very enthusiastic and motivated in many ways, and I loved working with her," Dr. Schrauwen said. 

Selected from a pool of 600 applicants, 45 high school, undergraduate and graduate students participated in the Helios Scholars at TGen internship this summer. In its ninth year, the Helios Scholars program has graduated close to 400 interns who have had hands-on, full-time, patient-focused research experiences at TGen.


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