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Chapter 5 - Genetic Models of Movement Disorders

from Section 1: - Basic Introduction

Published online by Cambridge University Press:  07 January 2025

By
Jing-Han Ng  and
Eng-King Tan
Edited by
Erik Ch. Wolters  and
Christian R. Baumann
Erik Ch. Wolters
Affiliation:
Universität Zürich
Christian R. Baumann
Affiliation:
Universität Zürich
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Summary

Various in-vitro (induced pluripotent stem cells–derived) and in-vivo genetic models (animal models like Caenorhabditis elegans, Drosophila melanogaster, zebrafish, rodents, and non-human primates) have been used to study movement disorders such as Parkinson’s disease, hereditary ataxia, Huntington’s disease, dystonia, and essential tremor. These genetic models have provided important clues on the underlying pathophysiologic mechanisms of these diseases and serve as useful platforms to unravel potential therapeutic targets. The next generation of genetic models is promising with the advancement of gene-editing techniques, such as CRISPR-Cas9, brain organoid technology, and identification of novel genes and loci from large-scale genetic studies will facilitate development of new genetic models.

Keywords

Genetic modelinduced pluripotent stem cellanimal modelbrain organoidgene editingmovement disordersGWAS
Type
Chapter
Information
International Compendium of Movement Disorders , pp. 74 - 84
Publisher: Cambridge University Press
Print publication year: 2025

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