Monozygotic twins discordant for a neurodegenerative disease provide a rare opportunity to delve into the genetic and non-genetic contributors to disease pathophysiology (See Sept 2014 news for a recent story on insights from twins discordant for C9ORF72 ALS). Using induced pluripotent stem cell (iPSC)-derived dopaminergic neurons from monozygotic twins discordant for Parkinson’s disease (PD), a team of researchers from the New York Stem Cell Foundation Research Institute have created a unique model of Parkinson’s disease (PD) in a dish. The study, published in the November 6 Cell Reports, revealed both genetic abnormalities common to both twins, such as the glucocerebrosidase (GBA) mutation, and unique characteristics of only the affected twins’ neurons, such as elevated levels of monoamine oxidase B (MAO-B) activity. The researchers were able to restore normal function of these cells by reducing MAO-B activity and increasing expression of GBA. Click here to read more about these discoveries and their therapeutic implications.
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