This technology is a HuB-targeting therapeutic approach that modulates 3′ UTR length, altering immunostimulatory dsRNA levels and downstream interferon signaling to treat neuroinflammatory and neurodegenerative diseases.
Neuroinflammatory and neurodegenerative diseases linked to disrupted RNA homeostasis, such as Aicardi-Goutières syndrome, lack effective, mechanism-based treatments that can safely reduce harmful, self-driven innate immune activation in neurons without undermining antiviral protection. Current care is largely supportive or relies on broad immunosuppression and anti-inflammatory strategies that do not correct the upstream source of aberrant immune triggering and can increase vulnerability to viral infection. This creates a need for more precise approaches to rebalance neuronal innate immune signaling, reducing pathological inflammation while preserving the necessary baseline antiviral immunity.
This technology is a method for reducing neuroinflammation by delivering an effector that decreases the activity of the neuron-enriched RNA-binding proteins HuB and/or HuC. Lower HuB/HuC activity shifts neuronal RNA processing, including 3′ UTR regulation, to reduce the formation of endogenous immunostimulatory double-stranded RNA species that can inappropriately trigger innate immune sensors. As a result, downstream type I interferon signaling and associated inflammatory stress in neurons are dampened, helping treat neuroinflammatory or neurodegenerative pathology.
This technology has been validated in neurons derived from human embryonic stem cells and the HEK293T cell line.
Patent Pending(US19/400,788)
IR CU23300, CU24306
Licensing Contact: Jerry Kokoshka
