PolyQ AR transcriptional altered activity and its role in SBMA pathogenesis
Carlo Rinaldi, Oxford University ($75,000 for one year)
We plan to elucidate polyQ AR transcriptional altered activity and its role in SBMA pathogenesis, filling a fundamental gap in the understanding of this disease, and to unravel the therapeutic mechanism of action of AR45, a naturally-occurring AR isoform able to fine-tune AR transcriptional activity. Ultimately our goal is to bring a gene therapy approach based on therapeutic delivery of AR45 into first-in-man clinical testing for SBMA patients.
