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SUMMARY:Rapid iPSC "Inclusionopathy" Models to Accelerate Diagnostic and T
 herapeutic Discovery in Synucleinopathies
DTSTART:20230323T100000
DTEND:20230323T110000
DTSTAMP:20260511T050356Z
UID:4b8239244785234535f30ff8a10a0b42871a41f1620006483de9c86a
CATEGORIES:Conferences - Seminars
DESCRIPTION:Prof. Vikram Khurana\, M.D.\, Ph.D.\, Ann Romney Center for Ne
 urologic Diseases at Brigham and Women’s Hospital\, Harvard Medical Scho
 ol\, Boston\, MA (USA)\nBIOENGINEERING SEMINAR\n \nAbstract:\nIn neurodeg
 enerative proteinopathies\, intracellular inclusions are histopathological
 ly and ultrastructurally heterogeneous but the significance of this hetero
 geneity is unclear. Patient- derived iPSC models\, while promising for dis
 ease modeling\, do not form analogous inclusions in a reasonable timeframe
  and suffer from limited tractability and scalability. In this talk\, I wi
 ll describe an iPSC toolbox that utilizes piggyBac-based or targeted trans
 genes to rapidly induce CNS cells with concomitant expression of misfoldin
 g-prone proteins. The system is scalable and amenable to screening and lon
 gitudinal tracking at single-cell and single-inclusion resolution. For pro
 of-of-principle\, cortical neuron alpha-synuclein inclusionopathy models h
 ave been engineered to form inclusions spontaneously or through exogenous 
 seeding by alpha-synuclein fibrils. These models recapitulated known fibri
 l- and lipid-rich inclusion subtypes in human brain\, shedding light on th
 eir formation and consequences. Genetic-modifier and protein-interaction s
 creens identified sequestered proteins in these inclusions\, including Rho
 A\, that were deleterious to cells when lost. Finally\, I will discuss how
  this new iPSC platform could facilitate biological\, diagnostic and thera
 peutic drug discovery for neurodegenerative proteinopathies.\n\n\nBio:\nDr
 . Khurana is Chief of the Division of Movement Disorders at Brigham and Wo
 men’s Hospital and Harvard Medical School. He is Principal Faculty at th
 e Harvard Stem Cell Institute\, and an Associate Member of the Broad Insti
 tute of Harvard and MIT. He is a member of the Board of Directors of the M
 SA Coalition. His clinical and research interests relate to neurodegenerat
 ive disorders focusing on Parkinson’s disease (PD) and related dementias
 \, rarer disorders including multiple system atrophy and ataxias.\nDr. Khu
 rana is a medical graduate of the University of Sydney\, Australia\, and c
 ame to Boston as a Fulbright Scholar in 2001\, obtaining his Ph.D. in neur
 obiology from Harvard University in 2006. He completed his neurology resid
 ency at Brigham and Women’s and Massachusetts General Hospitals\, and fe
 llowship in movement disorders and ataxia at Massachusetts General Hospita
 l\, where he was on the faculty from 2012 to 2016. Dr. Khurana received po
 stdoctoral training in the laboratories of Drs. Susan Lindquist and Rudolf
  Jaenisch at the Whitehead Institute for Biomedical Research (MIT)\, at wh
 ich time he led some of the first studies to identify and reverse patholog
 ies in human stem cells derived from PD patients.\nHis current research co
 ntinues to bring stem-cell technologies toward personalized and precise di
 agnostics and therapeutics for neurodegenerative disorders. Dr. Khurana’
 s research has been recognized through grants and awards\, including feder
 al grants from the NIH and Department of Defense\, foundation grants from 
 the American Academy of Neurology\, Michael J Fox Foundation\, Multiple Sy
 stem Atrophy Coalition\, National Ataxia Foundation\, Parkinson’s Diseas
 e Foundation among others. In 2018\, he was named a Robertson Investigator
  of the New York Stem Cell Foundation\, in 2019 a George C. Cotzias Fellow
  of the American Parkinson’s Disease Association and in 2020 an investig
 ator of the Aligning Science Across Parkinson’s Initiative.\n\n\nZoom li
 nk for attending remotely: https://epfl.zoom.us/j/61378478243
LOCATION:AI 1153 https://plan.epfl.ch/?room==AI%201153 https://epfl.zoom.u
 s/j/61378478243
STATUS:CONFIRMED
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