This technology is a method to modify Alfy protein levels to modulate levels of protein aggregation and clearance in the brain and other tissues to prevent, inhibit or treat a proteinopathy.
Neurodegenerative conditions such as Alzheimer's, Parkinson's, and Huntington's Disease stem from the abnormal accumulation of proteins within neurons. Under normal circumstances, cells utilize a cellular process known as aggrephagy to identify and disassemble these protein clusters. However, in the context of disease, neurons struggle to efficiently activate aggrephagy, leading to the build-up of toxic protein aggregates. Consequently, these neurons perish due to the harmful protein accumulation, giving rise to the symptoms associated with neurodegenerative diseases in affected individuals.
This technology identifies increasing aggrephagy protein Wdfy3 (Alfy) expression via either augmentation or a single base change in its DNA coding sequence, as a method of improving aggregate protein clearance from cells. To modify Alfy, isolated nucleic acid or viral vectors are administered. This alternative protein sequence also targets specific protein aggregates implicated in many neurodegenerative diseases, leading to neuroprotection. Neurodegenerative mouse models with intrastriatal injections with Alfy experienced delayed symptom onset and better survival rates. As such, augmenting Alfy may inhibit or treat protein aggregation diseases, particularly within the context of neurodegeneration.
This technology has been validated in mouse models and human-derived cells.
Patent Pending (WO/2023/091943)
IR CU22103, CU23286
Licensing Contact: Kristin Neuman
