Background pattern of a brain with neural connections
Michael Kaplitt

Michael Kaplitt

Lead PI (Core Leadership)

Weill Cornell Medicine

Michael G. Kaplitt, MD, PhD, is Professor and Vice-Chair for Research in the Department of Neurological Surgery at Weill Cornell Medicine. He received a PhD in molecular neurobiology from The Rockefeller University and an MD from Cornell University Medical College.

An international leader in gene therapy and functional neurosurgery for neurodegenerative disorders, he first described using adeno-associated virus in the mammalian brain in 1994., In 2003, he performed the first human gene therapy for Parkinson’s disease (PD)., and Dr. Kaplitt has also performed the first gene therapy for a genetic form of Parkinson’s disease and first induced pluripotent stem cell transplant for Parkinson’s disease, among many other translational innovations.

His lab research focuses upon using gene therapy to better understand and treat unmet needs in PD and other neuropsychiatric disorders. His many honors and awards include elected membership in the American Society for Clinical Investigation and the Society for Neurological Surgeon

Teams

Kaplitt

Themes

Circuitry and Brain-Body Interactions

Tags

Circuits and symptoms Alpha-synuclein Gut-brain axis Motor symptoms

Recent ASAP Preprints & Published Papers

AAV injection in the nodose ganglia of mouse

The gut-brain axis links the visceral organs to the medulla oblongata via the vagus nerve. Accessing to the afferent vagal pathway is important to dissect the role of cell populations in the bidirectional communication between brain and body. The jugular-nodose ganglion (JNG) complex contains the cell bodies of many heterogenous neural subpopulations responsible for sensing the physiological and pathological conditions of the thoracic and abdominal organs. However, the study of these ganglia is challenging in small animals due to size and location. Hence, in this protocol we describe a practical surgical approach to the vagal trunk, and the JNG complex in mice.

Phosphoglycerate kinase is a central leverage point in Parkinson’s Disease driven neuronal metabolic deficits

PAlthough certain drivers of familial Parkinson’s disease (PD) compromise mitochondrial integrity, whether metabolic deficits underly other idiopathic or genetic origins of PD is unclear. Here, we demonstrate that phosphoglycerate kinase 1 (PGK1), a gene in the PARK12 susceptibility locus, is rate limiting in neuronal glycolysis and that modestly increasing PGK1 expression boosts neuronal adenosine 5′-triphosphate production kinetics that is sufficient to suppress PARK20-driven synaptic dysfunction. We found that this activity enhancement depends on the molecular chaperone PARK7/DJ-1, whose loss of function significantly disrupts axonal bioenergetics. In vivo, viral expression of PGK1 confers protection of striatal dopamine axons against metabolic lesions. These data support the notion that bioenergetic deficits may underpin PD-associated pathologies and point to improving neuronal adenosine 5′-triphosphate production kinetics as a promising path forward in PD therapeutics.

Team Kaplitt

Michael Kaplitt

Michael Kaplitt

Lead PI (Core Leadership)

Per Svenningsson

Per Svenningsson

Co-PI (Core Leadership)

Roberta Marongiu

Roberta Marongiu

Co-PI (Core Leadership)

Ted Dawson

Ted Dawson

Co-PI (Core Leadership)

Jacquelyn Haytayan

Jacquelyn Haytayan

Project Manager

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