Annual Pediatric Science Day

Pediatric Science Day

The primary purpose of Pediatrics Science Day is to give trainees, whether students, post-docs, residents, fellows, or junior faculty, the opportunity to present their research to their colleagues, and to obtain scientific feed-back in a friendly environment. We strongly encourage the submission of abstracts that either have been recently presented at or have been submitted to national research meetings. However, most importantly, we would like to encourage the participation of all trainees in this unique opportunity!

In addition to abstract submissions, residents and fellows may also submit interesting clinical cases which include a brief case presentation, summary of unusual disease presentation or perplexing diagnosis, and a literature review of novel diagnostic methods and treatments.

2018 Guest Speaker

“Gene Delivery Translation in Spinal Muscular Atrophy”

Brian Kasper, PhD

Brian Kasper, PhD

Dr. Brian K. Kaspar is the founder and Chief Scientific Officer of Avexis, a gene therapy-based biotech company that was recently acquired by Novartis for $8.7 billion dollars. He has been a principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and Associate Professor in the Department of Pediatrics and Department of Neuroscience at The Ohio State University College of Medicine. In 2013, Dr. Kaspar was named Fellow of the American Association for the Advancement of Science (AAAS). He has published more than 100 scientific articles. He received his PhD in molecular pathology from UC San Diego and did his postdoctoral work on neuroscience and gene therapy at the Salk Institute.  Dr. Kaspar founded two biotech companies (Avexis and Milo) working on gene therapy.

The Kaspar Lab focuses on basic and translational studies related to neurological and neuromuscular disorders, particularly the mechanism(s) of neurodegeneration in Amyotrophic Lateral Sclerosis (ALS). Using rodent models of this disease, they investigate the involvement of various cell types in disease onset and progression. Furthermore, they are developing novel methods to deliver genes and therapies more efficiently to the nervous system and testing novel targets to combat this debilitating, lethal disease.

They are also investigating the biological control of embryonic and adult derived stem cells to evaluate cell cycle regulation and to develop methods to control cell differentiation to defined phenotypes, such as complex motor neurons.

Finally, they are developing muscle-enhancing strategies designed to combat musculoskeletal disorders. Follistatin is a potent antagonist of myostatin and enhances muscle size and mass when introduced to skeletal muscle by a one-time gene. Interestingly, they have shown that follistatin reduces the inflammatory environment in dystrophic muscles and induces improvements in functional strength in rodent models of Duchene Muscular Dystrophy. They are currently developing a human clinical trial for Inclusion Body Myositis with Dr. Jerry Mendell.

Disclosure: Dr. Kaspar has disclosed that he has the following relevant financial relationships. No one else in a position to control content has any financial relationship to disclose.

2018 Program Schedule

View the program schedule of events

Submitting an Abstract

Follow the Abstract Submission Guidelines and then complete the Abstract Submission Form and upload your abstracts by the Monday, May 21, 2018 at 11:59pm deadline.

Have Questions?

Contact Nikki Atzinger (aatzinger@ufl.edu).