Eric Wang PhD & Sub Subramony MD

PI/Investigators: Eric Wang, PhD & S. H. Subramony, MD, - University of Florida

Award type: Award for Innovative Mindset

Grant Title: Measurement of Frataxin mRNA in NHP and human biofluids

Summary: This project aims to develop biomarkers to detect early efficacy in clinical trials, by measuring frataxin in small vesicles present in the fluid that surrounds the brain and the spinal cord

Biomarkers play an important role to help determine optimal doses and provide early signs of potential efficacy in clinical trials testing gene therapy products. The investigators plan to measure the content of small vesicles found in the fluid surrounding the brain and spinal cord (cerebrospinal fluid or CSF), called exosomes. These vesicles are surrounded by membrane and contain protein, lipids and different types of RNA and that reflect the content of the cells in the brain that secrete them. The aim of the study is to develop methods to measure FXN mRNA in these vesicles found in CSF and blood of FA patients.