Shenoy Undergraduate Research Fellowship in Neuroscience (SURFiN)

One of the focuses of the Bradbury Lab at Nationwide Children’s Hospital is the development of a gene therapy for a rare, genetic, neurological disease called Vanishing White Matter disease (VWM). VWM is an autosomal recessive disorder that leads to ataxia, spasticity, seizures, neurodegeneration, and premature death in patients. PhD candidate and mentor, Jessica Herstine serves as the project lead and her thesis work has included the establishment of novel in vitro models such as patient-derived astrocytes to understand disease etiology. Furthermore, her NIH TL1 Training Grant includes the use of the I98M Toy mouse model to both characterize disease and to evaluate gene therapy efficacy. Currently, neonatal mice are being treated via intra-cerebrospinal fluid (intra-CSF) administration of numerous potential gene therapy constructs. Continual analysis of therapeutic efficacy is evaluated through behavior assays and magnetic resonance imaging (MRI) to visualize disease progression and myelin loss. Additional experiments include post-mortem analysis, to analyze histology, molecular disease markers, and vector biodistribution. Future studies include dose ranging studies and toxicology testing of the lead candidate gene therapy.

Our SURFiN fellow will assist with in life assessment of mice, as well as with molecular assays related to disease pathogenesis and potential attenuation with our therapies.

Information about our Lab and this project can be found here: https://www.simonsfoundation.org/surfin-program-lab-opportunities/