Neurodegenerative diseases are among the most pressing and devastating health challenges facing aging populations globally. We use stem cells to better understand and develop patient-specific therapies for these diseases. We are interested in Parkinson’s disease, multiple system atrophy and related disorders.
The Khurana Lab is located within the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital and Harvard Medical School. Together with our local, national and international collaborators, we strive to better understand and develop therapies for some of the most detrimental disorders of our time: neurodegenerative diseases caused by protein misfolding.
We focus on diseases caused by the mis-folding of specific proteins – alpha-synuclein, tau and ataxin proteins – that contribute hugely to neurodegenerative disease burden. These diseases include:
- synucleinopathies like Parkinson’s disease, multiple system atrophy and dementia with Lewy bodies
- tauopathies including progressive supranuclear palsy
- cerebellar ataxias including sporadic ataxias like multiple system atrophy, and hereditary ataxias like Friedreich’s ataxia and spinocerebellar ataxias
We generate induced pluripotent stem cells (iPSc) from patients with neurodegenerative disease to re-create the disease process “in a dish.” To better understand the consequences of protein misfolding in these cells, we introduce or genetically correct mutations in these cells with cutting-edge genome editing tools, including CRISPR-Cas9. We take a systems biology approaches to assess the cellular consequences of protein misfolding in these cells, and exploit these findings to drive biological experimentation, genomic studies and the development of targeted therapeutics.
We believe that, in time, insights from stem-cell-based models will enable us to match therapies to specific patients.
PROOF OF PRINCIPLE:
In 2013, working with Susan Lindquist and Rudolf Jaenisch at the Whitehead Institute, Vik Khurana and Chee Yeun Chung led an effort that succeeded in identifying and reversing abnormalities in stem-cell derived neurons from Parkinson’s disease patients (Chung*, Khurana* et al. Science, 2013). The approaches begun in that work are now being extended and broadened in the Khurana lab to encompass other diseases.
THE LAB AND CLINIC:
The Khurana lab is currently situated within the Ann Romney Center for Neurologic Disease and is part of the Harvard Stem Cell Institute.We are located in the spectacular Building for Transformative Medicine (BTM) in the main Longwood Medical Area of Harvard Medical School. BBF houses both the research and the clinical program, a proximity that facilitates our translational objectives. April 2018, Vik was appointed Chief of the Division of Movement Disorders at Brigham and Women’s Hospital.