Pan Li, Ph.D.

Headshot of Pan Li
  • Director of the Pathogenic Neurobiology Laboratory
  • Assistant Professor of Psychiatry and Behavioral Sciences

Research Interests

Spinocerebellar ataxia; Huntington's disease; and schizophrenia more



  • Director of the Pathogenic Neurobiology Laboratory
  • Assistant Professor of Psychiatry and Behavioral Sciences

Departments / Divisions



  • Ph.D.; Hong Kong University of Science and Technology (Hong Kong) (2010)

Research & Publications

Research Summary

My research focuses on the molecular pathogeneses of neurodegenerative disorders such as spinocerebellar ataxia types 2 and 12 (SCA2 and SCA12), and psychiatric disorders such as schizophrenia, with the goal of finding new therapeutic targets. Particular interest includes the molecular biology of disease loci containing expanded microsatellites, the contribution of antisense transcripts to pathogenesis, the role of RNA in neurotoxicity, exosome based drug delivery, and functional characterization of schizophrenia rare variants.

Selected Publications

Pan P. Li, Xin Sun, Guangbin Xia, Nicolas Arbez, Sharan Paul, Shanshan Zhu, H. Benjamin Peng, Christopher A. Ross, Arnulf H. Koeppen, Russell L. Margolis, Stefan M. Pulst, Tetsuo Ashizawa, Dobrila D. Rudnicki (2016) ATXN2-AS, a gene antisense to ATXN2, is associated with SCA2 and ALS. Annals of Neurology 80 (4), 600-615

Pan P. Li*, Russell L. Margolis (2021) Use of single guided Cas9 nickase to facilitate precise and efficient genome editing in human iPSCs. Scientific Reports 11: 9865 doi: 10.1038/s41598-021-89312-2. PMID: 33972655 (* corresponding author)

Hongxuan Feng, Qinshan Li, Russell L. Margolis, Pan P. Li* (2021) Generation of a human induced pluripotent stem cell line JHUi003-A with homozygous mutation for spinocerebellar ataxia type 12 using genome editing. Stem Cell Research 53: 102346 (* corresponding author)

Pan P. Li*, Roumita Moulick, Hongxuan Feng, Xin Sun, Nicolas Arbez, Jing Jin, Leonard O. Marque, Erin Hedglen, H.Y. Edwin Chan, Christopher A. Ross, Stefan M. Pulst, Russell L. Margolis, Sarah Woodson, Dobrila D. Rudnicki (2021) RNA toxicity and perturbation of rRNA processing in spinocerebellar ataxia type 2. Movement Disorders 36 (11), 2519-2529 (* corresponding author)

Qian Li, Yi Feng, Yingchao Xue, Xiping Zhan, Yi Fu, Gege Gui, Weiqiang Zhou, Jean-Philippe Richard, Arens Taga, Pan P. Li, Xiaobo Mao, Nicholas J. Maragakis and Mingyao Ying (2022) Edaravone activates the GDNF/RET neurotrophic signaling pathway and protects mRNA-induced motor neurons from iPS cells. Molecular Neurodegeneration 17(1):8

Activities & Honors


  • NARSAD Young Investigator’s Award, Brain & Behavior Research Foundation, 2014
  • Post-Doc Fellowship Award, National Ataxia Foundation, 2016
  • Postdoctoral Fellowship Award, Hereditary Disease Foundation, 2016 - 2018
  • Young Investigator-SCA Research Award, National Ataxia Foundation, 2018
  • Launch Award, Maryland Stem Cell Research Fund, 2021
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