Andrew Lieberman, M.D., Ph.D.

Michigan Neuroscience Institute Affliate
Gerald D. Abrams Collegiate Professor of Pathology
Director of Neuropathology
Department of Pathology and Clinical Laboratories
3510 MSRB I
1150 W. Medical Center Drive
Ann Arbor, MI 48109-5605


Dr. Andrew Lieberman is the Gerald D. Abrams Professor in the Department of Pathology and Director of Neuropathology at the University of Michigan. Dr. Lieberman received his BS from Duke University and his MD, Ph.D. from the University of Maryland Medical School. He completed residency training in Anatomic Pathology and fellowship training in Neuropathology at the University of Pennsylvania. Dr. Lieberman trained as a research fellow with Dr. Kenneth Fischbeck at the Neurogenetics Branch NINDS, NIH, and joined the University of Michigan Medical School faculty in 2001. He serves as Director of Neuropathology at the University of Michigan Medical Center and is Co-Director of the Michigan Protein Folding Diseases Initiative. Dr. Lieberman’s research has focused on the mechanism of neurodegeneration in inherited neurological disorders. The Lieberman Lab uses cell culture and mouse models to explore the pathogenesis of Kennedy’s disease, a polyglutamine expansion disorder, and Niemann-Pick C, a lysosomal storage disease resulting from impaired lipid trafficking.

Areas of Interest

The Lieberman Lab studies inherited neurodegenerative diseases to understand neurological dysfunction mechanisms to develop effective treatments. We are particularly interested in the CAG repeat diseases, a group of nine genetically related disorders caused by expanded CAG/glutamine tracts in the coding regions of disease-causing genes. Among these disorders is Kennedy disease, a degenerative disease that predominantly affects lower motor neurons and is caused by a mutation in the androgen receptor gene. The mutant protein misfolds, aggregates, and abnormally interacts with other proteins, leading to a toxic gain-of-function and a partial loss of normal function. We have developed both a knock-in mouse model and cell culture models of Kennedy disease. We are using these systems to understand how the mutant androgen receptor causes selective neuronal dysfunction that is characteristic of this disorder and to test novel therapeutic approaches.

Our laboratory also studies Niemann-Pick C, an autosomal recessive neurovisceral lipid storage disease with no cure. Mutations in two genes, Npc1 and Npc2, produce a clinically heterogeneous disorder characterized by devastating neurodegeneration that often begins in childhood. Loss of function mutations in either gene disrupts lipid trafficking and leads to glycosphingolipids' predominant accumulation within neurons. Mutations cause most cases of Niemann-Pick C in Npc1, a gene widely expressed in the brain. It is not known which cells are mechanistically involved in the neuropathology characteristic of this disease or whether the proteostasis machinery can be manipulated to achieve a functional rescue. We are using cellular and mouse models of Niemann-Pick C to address these questions and study the mechanisms leading to neuron death.

Published Articles or Reviews

Fbxo2 mediates clearance of damaged lysosomes and modifies neurodegeneration in the Niemann-Pick C brain. Liu EA, Schultz ML, Mochida C, Chung C, Paulson HL, Lieberman AP.JCI Insight. 2020 Oct 15;5(20):e136676.  

MEF2 impairment underlies skeletal muscle atrophy in polyglutamine disease. Nath SR, Lieberman ML, Yu Z, Marchioretti C, Jones ST, Danby ECE, Van Pelt KM, Sorarù G, Robins DM, Bates GP, Pennuto M, Lieberman AP.Acta Neuropathol. 2020 Jul;140(1):63-80.   

Hsp70 and Hsp40 inhibit an inter-domain interaction necessary for transcriptional activity in the androgen receptor. Eftekharzadeh B, Banduseela VC, Chiesa G, Martínez-Cristóbal P, Rauch JN, Nath SR, Schwarz DMC, Shao H, Marin-Argany M, Di Sanza C, Giorgetti E, Yu Z, Pierattelli R, Felli IC, Brun-Heath I, García J, Nebreda ÁR, Gestwicki JE, Lieberman AP, Salvatella X.Nat Commun. 2019 Aug 8;10(1):3562.  

Polyglutamine Repeats in Neurodegenerative Diseases. Lieberman AP, Shakkottai VG, Albin RL.Annu Rev Pathol. 2019 Jan 24;14:1-27.  

Targeting Hsp90/Hsp70-based protein quality control for treatment of adult onset neurodegenerative diseases. Pratt WB, Gestwicki JE, Osawa YLieberman AP.Annu Rev Pharmacol Toxicol. 2015;55:353-71.  

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