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NPJ Parkinsons Dis. 2016 Jun 2;2:16009. doi: 10.1038/npjparkd.2016.9. eCollection 2016.

Creation of a library of induced pluripotent stem cells from Parkinsonian patients.

Author information

1
Stem Cell Laboratory for CNS Disease Modeling, Wallenberg Neuroscience Center, Department of Experimental Medical Science, BMC A10, Lund University, Lund, Sweden.
2
Strategic Research Area MultiPark, Lund University, Lund, Sweden.
3
Lund Stem Cell Center, Lund University, Lund, Sweden.
4
Stem Cell Laboratory of Molecular Brain Research Group, Department of Neurobiology, A.I. Virtanen Institute, University of Eastern Finland, Kuopio, Finland.
5
Russian Academy of Sciences, Vavilov Institute of General Genetics, Moscow, Russia.
6
Department of Clinical Genetics and Biobanks, Office for Medical Services, Division of Laboratory Medicine, Lund, Sweden.
7
Parkinson Institute, Istituti Clinici di Perfezionamento, Milan, Italy.
8
Department of Neurobiology Research, Institute of Molecular Medicine, University of Southern Denmark, Odense, Denmark.

Abstract

Induced pluripotent stem cells (iPSCs) are becoming an important source of pre-clinical models for research focusing on neurodegeneration. They offer the possibility for better understanding of common and divergent pathogenic mechanisms of brain diseases. Moreover, iPSCs provide a unique opportunity to develop personalized therapeutic strategies, as well as explore early pathogenic mechanisms, since they rely on the use of patients' own cells that are otherwise accessible only post-mortem, when neuronal death-related cellular pathways and processes are advanced and adaptive. Neurodegenerative diseases are in majority of unknown cause, but mutations in specific genes can lead to familial forms of these diseases. For example, mutations in the superoxide dismutase 1 gene lead to the motor neuron disease amyotrophic lateral sclerosis (ALS), while mutations in the SNCA gene encoding for alpha-synuclein protein lead to familial Parkinson's disease (PD). The generations of libraries of familial human ALS iPSC lines have been described, and the iPSCs rapidly became useful models for studying cell autonomous and non-cell autonomous mechanisms of the disease. Here we report the generation of a comprehensive library of iPSC lines of familial PD and an associated synucleinopathy, multiple system atrophy (MSA). In addition, we provide examples of relevant neural cell types these iPSC can be differentiated into, and which could be used to further explore early disease mechanisms. These human cellular models will be a valuable resource for identifying common and divergent mechanisms leading to neurodegeneration in PD and MSA.

Conflict of interest statement

The authors declare no conflict of interest.

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