News Brief by Lushna Mehra
Scientists of The New York Stem Cell
Foundation (NYSCF) Research Institute used identical twins, an unrelated
Parkinson’s patient, and four healthy control subjects in order to create a
human stem cell disease model of Parkinson’s in a dish. Parkinson’s disease is deemed moderately
heritable, though the mode of inheritance involved is still unclear. DNA mutations that cause the production of the
enzyme glucocerebrosidase (GBA) are linked to a greater risk of developing
Parkinson’s in 30% of people with the mutation.
This low percentage shows that there are many other contributing factors
to the development of the disease. As a
result, identical twins both with the GBA mutation, though one had Parkinson’s
and one did not, were studied.
Scientists took skin samples from each of the twins to create the
cellular model of Parkinson’s by recreating dopamine deficiency and the
presence of alpha-synuclein, which collects when the disease is present. They noted that the affected twin’s neurons did
not have a strong neural network, produced less dopamine, and had higher levels
of the enzyme MAO-B. Treating such
neurons with molecules that lower MAO-B with excessive GBA was able to
normalize the alpha-synuclein and dopamine levels in cell models. This
indicates that some sort of combination therapy that targets the two enzymes
for the affected twin could be a possible treatment. More of such research could aid in
understanding the genetic inheritance of Parkinson’s and other related
disorders.
New York Stem Cell Foundation. "Scientists create Parkinson's disease in a
dish." ScienceDaily. ScienceDaily, 6 November 2014.
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