DPT students gain new insights on research, patient experience at unique muscular dystrophy conference

Dr. Claudia Senesac arranged for her students to talk with Duchenne advocates Dylan Miceli-Nelson, 15, and Austin Leclaire, 17. “I hope the PT students take away that this is a pretty serious disease and they are a big part of helping it,” Miceli-Nelson said.

In the gene therapy technique CRISPR-Cas9, researchers can target and snip out specific mutations in a genome, potentially eliminating mutations that cause genetic disease.

Science magazine called CRISPR-Cas9 technology 2015’s Breakthrough of the Year and it has shown promise for treating Duchenne muscular dystrophy in animal studies. UF Doctor of Physical Therapy students in the Pediatrics in Physical Therapy course heard an update on the research from Eric Olson, Ph.D., one of the leading scientists in the use of CRISPR-Cas9 for Duchenne, when they attended the 2016 Parent Project Muscular Dystrophy, or PPMD, Connect conference held this summer in Orlando.

The PPMD conference is unusual in that it brings together scientists, parents and Food and Drug Administration representatives to discuss advances in Duchenne therapies, said the students’ instructor Claudia Senesac, Ph.D., P.T., a clinical associate professor in the department of physical therapy.

“I think the most valuable lesson I learned during this conference is that every patient is unique and complex,” said DPT student Cassandra McGowan. “Ultimately we are working toward enhancing our patients’ quality of life and striving to increase their independence. We must remain sensitive, considerate and flexible with our treatments and relationships as we learn more about their challenges and goals.”