MAR 17, 2016 10:30 AM PDT

Promise and challenges of iPSC modeling in Parkinson's disease

Presented At Neuroscience
C.E. CREDITS: P.A.C.E. CE
Speakers
  • Associate Professor, Program Director of Gene Discovery and Stem Cell Modeling Parkinson's Institute and Clinical Center
    Biography
      Birgitt Schüle, MD, studied medicine at the Georg-August University, Göttingen and Medical University Lübeck, Germany from 1993-2001. She received her doctoral degree (Dr. med.) for her medical thesis in neurophysiology from the Georg-August University, Göttingen in 2001. Dr. Schüle worked during her neurological internship (2001-2002) at the Medical University Lübeck with Dr. Christine Klein on genetic forms of PD and dystonia. From 2003-2005, Dr. Schüle completed a postdoctoral fellowship in the laboratory of Dr. Uta Francke, Department of Genetics, Stanford University School of Medicine, before joining the Parkinson's Institute in October 2005.
      Dr. Schüle leads the genetics and biomarker program, since 2008, she has built a stem cell program developing novel "disease in-a-dish" models of Parkinson's disease using patient-derived induced pluripotent stem cells. In 2014, she became the director of the brain bank at the Parkinson's Institute.

    Abstract:

    Given the challenges of replicating Parkinson’s disease in animal models, returning to models that are human-based and highly clinically characterized may provide the most successful path forward. There is a critical need to differentiate Lewy body Parkinson’s disease from other non-Lewy body forms of parkinsonism. This is important because the underlying molecular pathomechanisms, and hence potential therapeutic targets, are unlikely to be similar for both.
    One exciting new avenue regarding modeling human disease is the use of induced pluripotent stem cell (iPSC) technology utilizing individual donors with clear clinical diagnosis of disease. In regard to modeling Lewy body Parkinson’s disease, patient-specific iPSCs lay the foundation for differentiation into the tissue-type of interest, i.e. mid-brain dopaminergic neurons. Using such a collection of iPSCs that is thoroughly characterized with high-quality and comprehensive clinical data and eventually with confirmative neuropathology at autopsy is the key for successful interpretation of findings.
     
    However, in order for iPSC modeling approach to produce validated results, several challenges must be addressed to reduce variability of intrinsic and extrinsic nature. This is why it is critical that the methods used to generate iPSCs are documented and performed in a consistent manner to minimize variability. Another critical area for improvement is the use of consistent (or at least well documented) methods for differentiation of these patient-derived iPSC lines into neuronal cultures. The field is in dire need of standardized and validated iPS laboratory practices to generate and characterize cells and phenotypes of interest to understand disease and to identify new targets.
     
    Learning objectives:
    •           Clinical and pathological characterization of Lewy body Parkinson’s disease
    •           Critical factors of intrinsic and extrinsic variability for iPS cell modeling
     


    Show Resources
    You May Also Like
    MAY 24, 2018 09:30 AM PDT
    C.E. CREDITS
    MAY 24, 2018 09:30 AM PDT
    DATE: May 24, 2018 TIME: 9:30PM PDT The current gold standard in in vitro pre-clinical cancer treatment screening remain cell lines,...
    APR 27, 2018 10:00 AM PDT
    C.E. CREDITS
    APR 27, 2018 10:00 AM PDT
    DATE: April 27, 2018TIME: 10:00am PST, 1:00pm ESTGlioblastoma (GBM) and Medulloblastoma (MB) are the most common adult and paediatric brain tumours, both of which can have devastating c...
    MAY 03, 2018 11:00 AM PDT
    MAY 03, 2018 11:00 AM PDT
    DATE: May 3, 2018TIME: 11:00AM PDT, 2:00PM EDTWhile stress is one of the leading causes of neuropsychiatric disorders, the molecular underpinnings of how stress induces alterations in b...
    MAY 22, 2018 08:00 AM PDT
    C.E. CREDITS
    MAY 22, 2018 08:00 AM PDT
    DATE: May 22, 2018TIME: 08:00AM PDT The nuclear receptors pregnane X receptor (PXR) and constitutive androstane receptor (CAR) are closely related transcription factors that...
    AUG 15, 2018 08:00 AM PDT
    C.E. CREDITS
    AUG 15, 2018 08:00 AM PDT
    DATE: August 15, 2018TIME: 08:00AM PDT, 11:00AM EDTThe failure of current chemotherapeutic strategies in the fight against cancer can be largely attributed to the occurrence of drug res...
    MAY 02, 2018 08:00 AM PDT
    C.E. CREDITS
    MAY 02, 2018 08:00 AM PDT
    Immunohistochemistry protocols, which utilize antibodies to visualize proteins in tissue sections, have many steps that need optimized to prevent non-specific background effects, artifacts, o...
    Loading Comments...