Exploring Blood based Biomarkers for Neurodevelopmental Diseases: Challenges and New Approaches
Aditi Bhattacharya, Reader(E)- Center for Brain Development and Repair, Institute for Stem Cell Biology and Regenerative Medicine
A significant limitation for neuropsychiatry is the
lack of reliable biomarkers for any disease condition that correlate well with
behavioral symptoms. For
instance, drug discovery efforts in ASD (especially FXS) have suffered setbacks
in trials because of a large placebo effect in behavioral tests. While
behavioral assessments are an indispensable part of any future trial and
medical practice, it is clear that no one measure will likely be able to track
all improvements and that a combination of behavioral assessments, with
unbiased molecular markers may have the required power. The current status of
research into molecular biomarkers suffers from several limitations including inaccessibility of the brain as sample tissue,
lack of substrate that is dynamic enough in the span of few weeks to accurately
represent changes in disease modifications in a trial setting and finally
limited clinical history for said marker is available. I will discuss using my
work with leveraging proteomic data derived from intact brain tissue to identify
blood based candidate proteins for the discovery of molecular barcodes in
Fragile X Syndrome, a leading genetic cause of intellectual disability and
Autism.
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