Vikram Khurana, M.D., Ph.D.
Harvard Medical School
The Khurana lab uses stem cells to better understand and develop patient-specific therapies for Parkinson’s disease, multiple system atrophy and related disorders.
Neurodegenerative diseases like Alzheimer’s and Parkinson’s disease are diseases caused when proteins abnormally fold and aggregate in specific cell types of the central nervous system. The Khurana lab, based in the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital, uses cutting-edge stem cell, genome-editing and in situ proteomic techniques to study how these hallmark protein-misfolding pathologies are related to genetic factors that predispose to these diseases, and how they may be reversed. The lab focuses in particular on Parkinson’s disease, multiple system atrophy, ataxias and related disorders.
Research in the Khurana lab addresses four central questions:
- How does protein misfolding perturb the physiology of and interaction between distinct cell types within the nervous system?
- How does the physiologic function of proteins relate to the toxicity that results when these proteins misfold?
- Do distinct forms (or “strains”) of misfolded proteins cause pathology in different ways?
- How can we best exploit our understanding of perturbed proteostasis for therapy?
Their combined approach encompasses tissue engineering, systems biology, unbiased genetic screens, protein interaction mapping, and mechanistic genetic studies. Their initial focus is on alpha-synuclein, a protein that misfolds in Parkinson’s disease (PD), dementia with Lewy bodies (DLB) and multiple system atrophy (MSA).
Vikram Khurana is on the faculty at Harvard Medical School, an attending neurologist at Brigham and Women’s Hospital and scientific co-founder of Yumanity Therapeutics. He is a medical graduate of the University of Sydney, Australia, and came to Boston as a Fulbright Scholar in 2001, obtaining his Ph.D. in neurobiology from Harvard University in 2006. He completed his residency in neurology at Brigham and Women’s and Massachusetts General Hospitals, and Fellowship training in movement disorders and ataxia at Massachusetts General Hospital. He received postdoctoral scientific training in the laboratories of Susan Lindquist and Rudolf Jaenisch at the Whitehead Institute, where he co-led a study that succeeded in identifying and reversing pathologies in stem cell models of Parkinson's disease (Chung*, Khurana* et al. Science 2013). Vik co-founded Yumanity Therapeutics in December 2014, a company that applies stem-cell technologies to drug discovery in neurodegenerative diseases (http://yumanity.com/about-leadership.php), and joined the Ann Romney Center and Harvard Stem Cell Institute in 2016.
Khurana, V.*, Tardiff, D. F., Chung, C. Y., & Lindquist, S.* (2015). Toward stem cell-based phenotypic screens for neurodegenerative diseases. Nature Reviews Neurology. PDF
Chung CY*, Khurana V*, Auluck PK, Tardiff DF, Mazzulli JR, Soldner F, Baru V, Lou Y, Freyzon Y, Cho S, Mungenast A, Muffat J, Mitalipova M, Pluth MD, Jui NT, Schüle B, Lippard SJ, Tsai LH, Krainc D, Buchwald, SL, Jaenisch R, Lindquist S. Identification and rescue of α-synuclein toxicity in Parkinson patient-derived neurons. Science. 2013 342 (6161): 983-87. *Equal contribution
Khurana V*o, Peng J*, Chung CY*, Auluck PK, Tardiff DF, Fanning S, Bartels T, Koeva M,Benyamini H, Lou Y, Nutter-Upham A, Tuncbag N, Baru V, Freyzon Y, Costanzo M, SanLuis B, Schöndorf DC, Barrasa MI, Caraveo G, Ehsani S, Sanjana N, Zhong Q, Gasser T, Vidal M, Deleidi M, Boone C, Berger B, Fraenkel E, Lindquist S. Genome-scale molecular networks link diverse neurodegenerative disease genes and processes to alpha-synuclein. Cell Systems 2017 4(2):157-170 *Equal contribution oCorresponding author
Chung CY*o, Khurana V*o, Loh K, Yi S, Sahni N, Hill D, Peng J, Vidal M, Ting A, Lindquist S*. In situproteome approaches connect alpha-synuclein directly to endocytic trafficking and mRNA metabolism. Cell Systems 2017 4(2):242-250 *Equal contribution oCorresponding author