This summer I had the opportunity to conduct research at the CHOP Abramson Pediatric Research Center under the direction of Dr. Adeline Vanderver. In particular, I assisted Dr. Akshata Almad with her project on developing an in-vitro model of H-ABC (hypomyelination with atrophy of the basal ganglia and cerebellum). H-ABC is a progressive leukodystrophy (disease related to white matter development) caused by a mutation in beta-tubulin which affects protein trafficking. Due to this, H-ABC is characterized by hypomyelination, delayed motor development, learning difficulties, and involuntary movements. The development of an in-vitro model of this disease, which does not currently exist, would be instrumental in not only understanding the cellular mechanisms of the pathology but also in testing various treatments. The model consisted of striatal neurons and oligodendrocytes differentiated from control and patient human induced pluripotent stem cell lines. By comparing the neurons and the oligodendrocytes between the control and patient lines, one would be able to comprehend exactly what this mutation affects as well as the efficacy of this model.
My experience in this lab has been two-fold. Not only have I been able to learn a myriad of lab skills, but also engage myself within the scientific process. Working with stem cells has allowed me to become familiar with tissue culture techniques ranging from feeding to passaging cells. I have also learned how to conduct immunohistochemistry staining, which is important for comparing the presence of proteins between the patient and control cells. Through gaining proficiency with ImageJ, I have been able to count stained cells as well as quantify the morphology of the oligodendrocytes through Sholl analysis. At the same time, I have been able to learn about how science is not exactly black and white. Often, the decisions that have to be made reside in more grey areas. Questions such as: should we let the cells grow for one more day? should we start differentiating them? etc. are ones that have no “right” answer. Rather the solutions are formulated from experience, other published papers, and scientific expertise. Overall, this research project has allowed me to immerse myself into neuroscience, strengthened my analytical and investigative skills, as well as inspired me to continue forth on a path of science and medicine.