Spinocerebellar Ataxia Type 1 Characteristics in Patient-Derived Fibroblast and iPSC-Derived Neuronal Cultures
Summary
Patient-derived cells recapitulate key pathological features of SCA1 pathogenesis providing a valuable tool for the identification of novel disease-specific processes. This model can be used for high throughput screenings to identify compounds, which may prevent or rescue neurodegeneration in this devastating disease. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
| Authors | Buijsen RAM, Hu M, Sáez-González M, Notopoulou S, Mina E, Koning W, Gardiner SL, van der Graaf LM, Daoutsali E, Pepers BA, Mei H, van Dis V, Frimat JP, van den Maagdenberg AMJM, Petrakis S, van Roon-Mom WMC |
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| Journal | Movement disorders : official journal of the Movement Disorder Society |
| Publication Date | 2023 Aug;38(8):1428-1442 |
| PubMed | 37278528 |
| DOI | 10.1002/mds.29446 |