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December 1, 2014
Parkinson’s disease is a progressive movement disorder involving the malfunction and death of neurons in the brain. Symptoms of the disease include tremors, slowed movement, trouble walking and changes in speech. Despite the abundance of animal models that have been generated to study Parkinson’s disease, there is no cure for the disorder. This week’s Science First looks at another innovative “disease in a dish” model which does not rely upon animal use—this one aimed at better understanding the underlying mechanisms of Parkinson’s disease. In order to better understand the genetic and non-genetic risk factors that contribute to Parkinson’s disease, researchers at The New York Stem Cell Foundation Research Institute generated human-relevant models of the disorder from a pair of identical twins–one who was affected by Parkinson’s and the other who was unaffected. Researchers also made cellular models of the disease from healthy control subjects and another individual affected by the disorder. Skin cells from patients were reprogrammed into induced pluripotent stems cells which were then differentiated into the specific kinds of neurons affected by the disorder. These cells mimicked some of the key pathological features of Parkinson’s disease, including the accumulation of a protein called α-synuclein and a reduction in the ability to make and release dopamine, an important chemical messenger in the brain. Interestingly, comparisons of these models at the molecular level revealed important differences between the affected and unaffected twin, suggesting some factors that may have contributed to the onset of Parkinson’s disease in the affected individual, and revealing possible targets for disease-modifying therapies. What are your thoughts on disease in a dish models for human conditions? Send your questions and comments to sciencecorner@navs.org. I look forward to hearing from you. –Dr. Pam Osenkowski, Director of Science Programs |
Scientists create Parkinson’s disease in a dish November 6, 2014A team of scientists led by The New York Stem Cell Foundation (NYSCF) Research Institute successfully created a human stem cell disease model of Parkinson’s disease in a dish. For more information see: Science Daily Click here for access to the Cell Reports paper describing this exciting in vitro model. |