scholarly journals Motor neuron derivation from human embryonic and induced pluripotent stem cells: experimental approaches and clinical perspectives

2014 ◽  
Vol 5 (4) ◽  
pp. 87 ◽  
Author(s):  
Irene Faravelli ◽  
Monica Bucchia ◽  
Paola Rinchetti ◽  
Monica Nizzardo ◽  
Chiara Simone ◽  
...  
Keyword(s):  
Stem Cells ◽  
Motor Neuron ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Experimental Approaches ◽  
Induced Pluripotent

Effective motor neuron differentiation of hiPSCs on a patch made of crosslinked monolayer gelatin nanofibers

2016 ◽  
Vol 4 (19) ◽  
pp. 3305-3312 ◽  
Author(s):  
Yadong Tang ◽  
Li Liu ◽  
Junjun Li ◽  
Leqian Yu ◽  
Francesco Paolo Ulloa Severino ◽  
...  
Keyword(s):  
Stem Cells ◽  
Motor Neuron ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Electrode Array ◽  
Plug And Play ◽  
Neuron Differentiation ◽  
Neuron Spike ◽  
Induced Pluripotent ◽  
Multi Electrode Array

A patch made of crosslinked monolayer nanofibers was used for motor neuron differentiation from human induced pluripotent stem cells and plug-and-play with a commercial multi-electrode array for neuron spike recording.

Human motor neuron generation from embryonic stem cells and induced pluripotent stem cells

2010 ◽  
Vol 67 (22) ◽  
pp. 3837-3847 ◽  
Author(s):  
M. Nizzardo ◽  
C. Simone ◽  
M. Falcone ◽  
F. Locatelli ◽  
G. Riboldi ◽  
...  
Keyword(s):  
Stem Cells ◽  
Embryonic Stem Cells ◽  
Motor Neuron ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Embryonic Stem ◽  
Human Motor ◽  
Induced Pluripotent

scholarly journals Motor Neuron Generation from iPSCs from Identical Twins Discordant for Amyotrophic Lateral Sclerosis

Cells ◽  
2020 ◽  
Vol 9 (3) ◽  
pp. 571 ◽  
Author(s):  
Emily R. Seminary ◽  
Stephanie Santarriaga ◽  
Lynn Wheeler ◽  
Marie Mejaki ◽  
Jenica Abrudan ◽  
...  
Keyword(s):  
Stem Cells ◽  
Amyotrophic Lateral Sclerosis ◽  
Motor Neuron ◽  
Induced Pluripotent Stem Cells ◽  
Motor Neurons ◽  
Pluripotent Stem Cells ◽  
Identical Twins ◽  
Sporadic Als ◽  
Induced Pluripotent ◽  
Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a complex neurodegenerative disorder characterized by the loss of the upper and lower motor neurons. Approximately 10% of cases are caused by specific mutations in known genes, with the remaining cases having no known genetic link. As such, sporadic cases have been more difficult to model experimentally. Here, we describe the generation and differentiation of ALS induced pluripotent stem cells reprogrammed from discordant identical twins. Whole genome sequencing revealed no relevant mutations in known ALS-causing genes that differ between the twins. As protein aggregation is found in all ALS patients and is thought to contribute to motor neuron death, we sought to characterize the aggregation phenotype of the sporadic ALS induced pluripotent stem cells (iPSCs). Motor neurons from both twins had high levels of insoluble proteins that commonly aggregate in ALS that did not robustly change in response to exogenous glutamate. In contrast, established genetic ALS iPSC lines demonstrated insolubility in a protein- and genotype-dependent manner. Moreover, whereas the genetic ALS lines failed to induce autophagy after glutamate stress, motor neurons from both twins and independent controls did activate this protective pathway. Together, these data indicate that our unique model of sporadic ALS may provide key insights into disease pathology and highlight potential differences between sporadic and familial ALS.

scholarly journals The Potential of Induced Pluripotent Stem Cells to Test Gene Therapy Approaches for Neuromuscular and Motor Neuron Disorders

2021 ◽  
Vol 9 ◽  
Author(s):  
Marisa Cappella ◽  
Sahar Elouej ◽  
Maria Grazia Biferi
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Motor Neuron ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Molecular Mechanisms ◽  
Viral Vector ◽  
Patient Specific ◽  
Major Advance ◽  
Induced Pluripotent

The reprogramming of somatic cells into induced pluripotent stem cells (iPSCs) represents a major advance for the development of human disease models. The emerging of this technique fostered the concept of “disease in a dish,” which consists into the generation of patient-specific models in vitro. Currently, iPSCs are used to study pathological molecular mechanisms caused by genetic mutations and they are considered a reliable model for high-throughput drug screenings. Importantly, precision-medicine approaches to treat monogenic disorders exploit iPSCs potential for the selection and validation of lead candidates. For example, antisense oligonucleotides (ASOs) were tested with promising results in myoblasts or motor neurons differentiated from iPSCs of patients affected by either Duchenne muscular dystrophy or Amyotrophic lateral sclerosis. However, the use of iPSCs needs additional optimization to ensure translational success of the innovative strategies based on gene delivery through adeno associated viral vectors (AAV) for these diseases. Indeed, to establish an efficient transduction of iPSCs with AAV, several aspects should be optimized, including viral vector serotype, viral concentration and timing of transduction. This review will outline the use of iPSCs as a model for the development and testing of gene therapies for neuromuscular and motor neuron disorders. It will then discuss the advantages for the use of this versatile tool for gene therapy, along with the challenges associated with the viral vector transduction of iPSCs.

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