PiN Faculty Member - Clemens Scherzer, MD

Clemens Scherzer, MD

Associate Professor of Neurology

Laboratory for Neurogenomics
65 Landsdowne Street
Cambridge, MA 02139
Tel: 617-768-8427
Fax: 617-768-8520
Visit my lab page here.

Our goal is to decipher three dimensions of the transcriptional code of Parkinson’s - transcript abundance, splice structure, and sequence - and the mechanisms by which transcriptional information is compromised and perturbs biological systems and phenotypes. While the number of human genes has shrunken to ~22,000, an ever-increasing number of >100,000 unique isoforms and non-protein coding, regulatory RNAs may account for the complexity of the human brain in health and disease. We hypothesize that genetic, epigenetic, and environmental risk factors cause disease through integrated modulation of RNA abundance, sequence, and splicing. We use massively parallel whole transcriptome sequencing, computation, molecular, and systems biology to break this RNA code and strive to rapidly translate insights into new diagnostics and medicines.

This innovative approach has deciphered critical bits of transcriptional information on onset mechanism, novel therapeutic targets, and causal transcripts. It revealed that reduced expression of the bioenergetics genes controlled by a master regulator - PGC-1alpha - likely occurs during the initial stages of disease, long before the onset of symptoms, and identified a molecular switch to turn off α-synuclein, the protein that accumulates in Parkinson’s patients.

Because we believe that transformative discovery requires transcending conventional research boundaries, the Scherzer laboratory is interdisciplinary, including neuroscientists, genomics, computational, as well as clinical scientists. Our longitudinal Harvard NeuroDiscovery Center Biomarker Study and Biobank of 2,000 participants serves as express lane for translating lead RNAs into biomarkers and for developing a future personalized neurology. Highthroughput drug screens are used to translate transcriptional targets into new therapies.

Last Update: 9/16/2020


For a complete listing of publications click here.



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