Assessment of Neurodegeneration Reversal in a Spinocerebellar Ataxia Type 1 (SCA1) Mouse Model by 1H MRS at 9.4 Tesla

Gulin Oz¹, H. Brent Clark¹, Christopher D. Nelson¹, Dee M. Koski¹, Manda L. Vollmers¹, Harry T. Orr¹

To assess the sensitivity of high field ¹H MRS to reversal of neurodegeneration, we utilized a conditional mouse model of SCA1. We turned off the mutated gene from 12-24 weeks and measured cerebellar neurochemical profiles of treated and untreated conditional mice and controls at 9.4T using short echo LASER and LCModel. The altered levels of multiple biomarkers partially reversed to control values at 24 weeks in the treated mice, in agreement with partial reversal of Purkinje cell pathology. These data demonstrate the ability of MRS to non-invasively detect disease reversal and its potential utility in future pre-clinical and clinical trials.