Development of a new cell model of Huntington's disease to evaluate the effectiveness of potential drugs at preclinical stages
Nina Kraskovskaya, Polina Parfionova, Natalia Mikhailova · FENS Forum 2024
Huntington's disease (HD) is an inherited, incurable neurodegenerative disease that primarily affects neurons in the striatum. In the present study, we successfully used an optimized cell model of HD [Kraskovskaya et al., 2023] to evaluate the effectiveness of the sigma 1 receptor agonist compound 3-PPP (N-n-propyl-3-(3-hydroxyphenyl)piperidine). We showed that striatal neurons obtained by direct reprogramming of dermal fibroblasts from HD patients (HD iSN) exhibit aggregates of mutant huntingtin, but the number of cells containing aggregates is less than 5% of the total. We also showed that t
ePoster · Neuroscience