Advantages:
- Reduction in Pathology
- Improved Memory and Cognitive Function
- Targeted Delivery and Brain-wide Distribution
Business Summary:
Alzheimer’s disease (AD) is a neurodegenerative disease that involves the accumulation of A-Beta proteins and Tau proteins within the brain and leads to cognitive impairments. The accumulation of these proteins can occur in various parts of the brain, causing a decline in synaptic plasticity. The ubiquitin ligase 3A (UBE3A) protein, which is critical for normal synaptic function, is reduced in AD. UBE3A gene therapy in a mouse model of tauopathy increased the expression of the UBE3A protein, decreased tau pathology, and rescued cognitive deficits.
Western blot analysis of hippocampus soluble fraction from non-transgenic (NTG), AAV9-GFP treated Tg4510, and AAV9-StuB treated Tg4510 mice. Panels A and D show micrographs of protein bands for total tau (H150), phosphorylated tau (AT180 and pSer396), and total protein. Panel B presents quantification of band intensity for H150, AT180 (Panel C), and pSer396 (Panel D). Statistical analysis using a student's t-test indicated a significant reduction in pSer396 levels in AAV9-StuB treated mice compared to AAV9-GFP treated mice (*p < 0.05, ***p < 0.001). These results suggest the potential of AAV9-StuB treatment to decrease tau pathology in Alzheimer's disease.
Desired Partnerships:
- License
- Sponsored Research
- Co-Development