Formation of Functional Motor units and Recovery from Paralysis Using Embryonic Stem Cell Transplantation

The technology describes the potential of embryonic stem cell-derived motor neurons to functionally replace those destroyed in a paralyzed rat model. JHU researchers have successfully demonstrated the ability to increase the success of transplanted axons extending out of the spinal cord into ventral roots using motor neurons derived from embryonic stem (ES) cells. Furthermore, transplant-derived axons reached muscle, formed neuromuscular junctions, were physiologically active, and mediated partial recovery from paralysis. Description (Set) Johns Hopkins University is seeking licensees for an innovative method to restore functional neuromuscular junctions by transplanting embryonic stem cell-derived early motor neurons. Trauma, autoimmune disorders, neurodegenerative diseases cause debilitating nerve damage and paralysis. Stem cell transplants have great potential for successful therapeutics. However, multiple obstacles exist to achieving sustained cell survival, growth and function. Challenges specific to neuronal cell transplant include inhibitory effects of myelin on nascent axon outgrowth and ability to establish functional muscle cell junctions. Survival and growth of early neurons are dependent on a balance of many chemical signaling factors which to date has not been elucidated. JHU scientists have uncovered a specific combination of factors which induce early neurons derived from embryonic stem (ES) cells to mature and extend axons to host skeletal muscle cells and restore function in animal models of paralysis. Advantages:
• Treatment of transplanted stem cell-derived early neurons with a cAMP agonist boosts survival and maturity of cells to increase numbers of available maturing neurons able to restore neuromuscular connections.
• Addition of a phosphodiesterase inhibitor reduces inhibitory effects of myelin to maximize axon outgrowth and enervate more muscle tissue that lacks neuronal connections and increase muscle function.
• Introducing an axon trophic factor attracts growing axons toward muscle tissue to form functional neuromuscular junctions that can restore physiological and behavioral function in paralyzed tissue. Proposed Use (Set) This therapeutic transplant strategy could be developed as a cell-based therapeutic for traumatic nerve injury, degenerative motor neuron diseases and autoimmune nervous system disorders.

Inventor(s): Kerr, Douglas

Type of Offer: Licensing

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