This technology is a nucleotide sequence for amplifying the expression of WDFY3 gene or its encoded protein, Autophagy-Linked FYVE (ALFY), that can be used to promote protein aggregate clearance in neurodegenerative diseases.
Many adult-onset neurodegenerative diseases, such as Alzheimer’s, Huntington’s, and Parkinson’s diseases, share the pathological feature of misfolded protein aggregates in the brain. Current methods to treat neurodegenerative diseases have been targeting these aggregates. However, their efficacy has been limited in both reducing the aggregates and slowing disease progression. Neurons have the innate ability to selectively degrade protein aggregates with macroautophagy; activating this inherent mechanism can enable more effective clearance of aggregates and treatment of neurodegenerative diseases.
This technology is a nucleotide sequence that activates the degradation of pathogenic protein aggregates by increasing the levels of Autophagy-Linked FYVE (ALFY) protein, encoded by the gene WDFY3. Universally expressed in the brain, ALFY is an adapter protein critical for the selective clearance of protein aggregates via the autophagy pathway. ALFY levels are increased by administering the truncated or full-length long-coding RNA WDFY3-AS2. Upregulating WDFY3-AS2 augments the expression of WDFY3 and ALFY. In mouse models of Huntington’s, Parkinson’s, and Alzheimer’s diseases, overexpressing ALFY decreased protein aggregate levels in the brain and protected against disease pathogenesis.
Patent Pending (WO/2024/233957)
IR CU23286
Licensing Contact: Kristin Neuman