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
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
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
"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