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Review

Stem Cell Models and Gene Targeting for Human Motor Neuron Diseases

1
Department of Biomedical Sciences, University of Illinois College of Medicine, Rockford, IL 61107, USA
2
Department of Bioengineering, University of Illinois at Chicago, Chicago, IL 60607, USA
*
Author to whom correspondence should be addressed.
Academic Editors: Leonidas A. Phylactou and Andrie Koutsoulidou
Pharmaceuticals 2021, 14(6), 565; https://0-doi-org.brum.beds.ac.uk/10.3390/ph14060565
Received: 29 April 2021 / Revised: 5 June 2021 / Accepted: 8 June 2021 / Published: 12 June 2021
(This article belongs to the Special Issue Neuromuscular Disorders: Current Gene and Cell Therapeutic Approaches)
Motor neurons are large projection neurons classified into upper and lower motor neurons responsible for controlling the movement of muscles. Degeneration of motor neurons results in progressive muscle weakness, which underlies several debilitating neurological disorders including amyotrophic lateral sclerosis (ALS), hereditary spastic paraplegias (HSP), and spinal muscular atrophy (SMA). With the development of induced pluripotent stem cell (iPSC) technology, human iPSCs can be derived from patients and further differentiated into motor neurons. Motor neuron disease models can also be generated by genetically modifying human pluripotent stem cells. The efficiency of gene targeting in human cells had been very low, but is greatly improved with recent gene editing technologies such as zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN), and CRISPR-Cas9. The combination of human stem cell-based models and gene editing tools provides unique paradigms to dissect pathogenic mechanisms and to explore therapeutics for these devastating diseases. Owing to the critical role of several genes in the etiology of motor neuron diseases, targeted gene therapies have been developed, including antisense oligonucleotides, viral-based gene delivery, and in situ gene editing. This review summarizes recent advancements in these areas and discusses future challenges toward the development of transformative medicines for motor neuron diseases. View Full-Text
Keywords: human pluripotent stem cells; motor neuron diseases; gene editing; neurodegeneration; axonopathy; amyotrophic lateral sclerosis; spinal muscular atrophy; gene therapy; antisense oligonucleotides; viral vectors human pluripotent stem cells; motor neuron diseases; gene editing; neurodegeneration; axonopathy; amyotrophic lateral sclerosis; spinal muscular atrophy; gene therapy; antisense oligonucleotides; viral vectors
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MDPI and ACS Style

Karpe, Y.; Chen, Z.; Li, X.-J. Stem Cell Models and Gene Targeting for Human Motor Neuron Diseases. Pharmaceuticals 2021, 14, 565. https://0-doi-org.brum.beds.ac.uk/10.3390/ph14060565

AMA Style

Karpe Y, Chen Z, Li X-J. Stem Cell Models and Gene Targeting for Human Motor Neuron Diseases. Pharmaceuticals. 2021; 14(6):565. https://0-doi-org.brum.beds.ac.uk/10.3390/ph14060565

Chicago/Turabian Style

Karpe, Yashashree, Zhenyu Chen, and Xue-Jun Li. 2021. "Stem Cell Models and Gene Targeting for Human Motor Neuron Diseases" Pharmaceuticals 14, no. 6: 565. https://0-doi-org.brum.beds.ac.uk/10.3390/ph14060565

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