Engineering Human Skeletal Muscle
- Description: I work in Dr. Bursac’s lab to develop human muscle that can regenerate after injuries. I also utilize tissue engineering strategies to create models of glycogen storage diseases that can serve as a testing platform for experimental treatment. Over the summer of 2018, I worked on my research projects as a REU scholar at Duke and won an award for my poster entitled “Bioengineered Human Skeletal Muscle as an Emerging Model for Pompe Disease”. In October 2018, I presented my findings as a platform presentation at BMES, the largest biomedical engineering conference in the nation. We are currently working on publishing our results. I described my work in more detail in my GCS thesis. The abstract is included here.
- Pompe disease is a rare lysosomal storage disorder characterized by deficiencies in acid-alpha glucosidase (GAA). Despite ongoing research into the pathophysiology of the disease and attempts to develop new treatments, the underlying disease mechanism is not completely understood. Through my research in the Bursac lab, I was involved with a project attempting to engineer an improved model of Pompe Disease using tissue engineering. We engineered a 3D human skeletal muscle model using primary myoblasts derived from diseased and healthy patients. Our model recapitulated structural phenotypes of Pompe Disease such as lysosomal enlargement, functional phenotypes such as increased susceptibility to metabolic stress, and biochemical phenotypes such as elevated glycogen levels. We further treated our model with standard enzymatic replacement therapy and observed glycogen clearance. Our model can serve as an in vitro screening tool for identifying novel medicines for Pompe Disease. As a proof of concept, we treated engineered skeletal muscle with adeno-associated virus encoding hGAA to assess its therapeutic potential. Taken together, our work showcases the utility and versatility of engineered skeletal muscle tissues, both as a discovery tool to further our understanding of fundamental biology and as a powerful validation tool furthering the quest of engineer better medicines.
- Relation to GC focus: tissue engineering attempts to replicate the complex interactions present in biological tissue in carefully controlled laboratory settings. The muscle engineering work I’m involved in can potentially lead to improved treatments for a wide range of muscle wasting diseases and improve quality of life for millions of patients. It can either become a therapeutic itself or lead to the development of better treatment options for patients.
- Start date: October 2016
- End date: May 2020
- Total time committed to date: 8 semesters + 3 months over the summer
- Hours: 1000+
Implementing Precision Medicine: Family Health History
- I worked in a team of 6 students in a project team headed by Dr. Susanne Haga and Dr. Lori Orlando of Duke School of Medicine. We were interested in studying the role of family history collection on improving preventative care. I decided to join this project because it was completely different the wet lab research I previously worked on. This was considered clinical research, and we ran an IRB approved study during the year. The research topic was also more related to social sciences but in the context of medicine. Our main research objective was to study the perceptions and barriers to implementation for a new family health history (FHH) collection platform developed by Dr. Orlando at Duke, called MeTree.
We kicked off our fall semester by conducting a literature review on current family health history collection tools as well as perceptions, attitudes, and barriers to collecting FHH. Over winter break, we designed a study focused on investigating the attitudes and perceptions of FHH within the young adult population. We launched an online survey targeting Duke students and young adults in the Durham community the following spring. In addition to questions assessing current knowledge, behaviors, and perceived importance of FHH, we also produced an educational video (starring Dr. Orlando and other Duke faculty members) to see whether written material or video could promote knowledge of FHH. I was heavily involved in drafting survey questions, programming questions onto Qualtrics, launching the survey, recruiting participants, and analyzing the final results.
We presented our final results in a poster format at the bass connections symposium at the end of the school year. Luckily, our manuscript also got accepted in Plos One last year. I was involved in the revision process as well. Read the full paper here.
- Relation to GC focus: Overall, this experience enabled me to identify key barriers in the implementation of novel medical technologies beyond science itself. It also broadened my definition of my GCS challenge – Engineering Better Medicines. Medicine takes many forms, and preventative medicine is certainly an important aspect of medicine that has great potential to improve patient outcomes. Our findings highlight the critical roles social and economic factors play in medicine. It’s not always about scientific details.
- Start date: August 2017
- End date: May 2018
- Hours: 150
- Read out published paper here https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0224283