Mary Jia presenting her poster at the ASGCT 2022 conference in Washington D.C.!

Author: Mary Jia | Major: Biomedical Engineering | Semester: Spring 2022

In early May of 2022, I attended the American Society for Gene and Cell Therapy (ASGCT) conference in Washington, D.C. along with my lab colleagues to present our research progress. It was the first conference we had ever attended for our lab in person, which was a barrier made insurmountable by the dreaded COVID-19 pandemic in past years. The primary purpose of ASGCT is for biotech companies and academic labs to present their work for other scientists and students in the gene and cell therapy field.

I have been working in the Nelson lab since my freshman year developing exon skipping prime editors for Duchenne Muscular Dystrophy (DMD).  DMD is an X-linked monogenic muscle wasting genetic disease that is caused by mutations in the DMD gene producing a non-functional dystrophin protein. Because deletions in the exons of DMD that disrupt the reading frame are often the cause of DMD, skipping or excising whole exon regions are often frame-restoring and can result in phenotype recovery. I was presenting our preliminary successful prime editor exon skipping results in mouse myoblast cell lines.

At the conference, I attended many talks by researchers working on the cutting edge of prime editor development and optimization to increase editing efficiency and specificity, including those that had developed prime editor itself. Additional talks by those specializing in the field of ethics as it relates to gene and cell therapy were incredibly enlightening. Francis Collins, the new science advisor to President Biden and former NIH director had also provided an excellent talk on the future of our field and the direction we need to emphasize moving forward.

After returning from the conference, I feel a renewed sense of motivation and clarity in terms of the experiments I will need to conduct in the future. Although it was initially intimidating to present my work in the presence of scientists of great renown in my field, I am so grateful that I was given the opportunity to attend this conference and expand my perspective of the gene editing field. This experience will continue to fuel my motivation to further advance my field and continue learning new techniques or methods. Furthermore, I am now more than ever certain that I will continue working within this field in graduate school and, hopefully, as an eventual biomedical engineering professor.