Michael Kaplitt, M.D., Ph.D.
Director, Laboratory of Molecular Neurosurgery, Weill Cornell Medical College
Under the direction of Dr. Michael Kaplitt, goals are to improve motor function in Parkinson’s disease as well as reduce the adverse effects of levadopa(L-dopa) therapy. Dr. Kaplitt and his team of scientists are examining the effects of a key protein, p11, which regulates brain signaling by the serotonin neurotransmitter which, in turn, appears to modulate signaling of dopamine. Dr. Kaplitt and his teamhave shown that inhibiting or blocking p11 improves motor function in mouse models of Parkinson’s disease as well as in normal mice under challenging conditions. The team accomplished this by altering the genetic expression of p11 using a technique common to gene therapy: gene delivery via an adeno-associated virus (a virus similar to the one that causes the common cold).
Because they have already used AAV-mediated gene therapy in human patients with Parkinson’s disease, their findings on p11 expression could have huge therapeutic implications.