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Conversion of Human Induced Pluripotent Stem Cells iPSCs into Functional Spinal and Cranial Motor Neurons Using PiggyBac Vectors
Published on: May 1, 2019
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Stem Cell Models and Gene Targeting for Human Motor Neuron Diseases
Yashashree Karpe1, Zhenyu Chen1,2, Xue-Jun Li1,2
1Department of Biomedical Sciences, University of Illinois College of Medicine, Rockford, IL 61107, USA.
Pharmaceuticals (Basel, Switzerland)
|July 2, 2021
Summary
Advancements in gene editing and stem cell technology enable the creation of motor neuron disease models. These models aid in understanding disease mechanisms and developing targeted gene therapies for conditions like ALS.
Area of Science:
- Neuroscience
- Stem Cell Biology
- Genetics
Background:
- Motor neuron degeneration causes debilitating neurological disorders such as amyotrophic lateral sclerosis (ALS).
- Induced pluripotent stem cell (iPSC) technology allows derivation and differentiation of patient-specific motor neurons.
- Gene editing technologies have significantly improved targeting efficiency in human cells.
Purpose of the Study:
- To review recent advancements in stem cell-based motor neuron disease models.
- To explore the application of gene editing tools in understanding motor neuron diseases.
- To summarize progress in targeted gene therapies for motor neuron disorders.
Main Methods:
- Utilizing induced pluripotent stem cells (iPSCs) from patients.
- Generating motor neuron disease models through genetic modification of pluripotent stem cells.
- Applying gene editing technologies like CRISPR-Cas9, ZFNs, and TALENs.
- Developing targeted gene therapies including antisense oligonucleotides and viral gene delivery.
Main Results:
- Human iPSC-derived motor neurons provide valuable models for studying neurological disorders.
- Gene editing tools have revolutionized the efficiency of genetic modification in stem cells.
- Targeted gene therapies show promise for treating motor neuron diseases.
Conclusions:
- The combination of stem cell models and gene editing offers powerful tools for dissecting disease pathogenesis.
- Significant progress has been made in developing gene therapies for motor neuron diseases.
- Further research is needed to overcome challenges and develop transformative medicines.
