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Gene therapy target validation

Modelling efficacy and mechanism
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Model for gene therapy drug discovery

The use of induced pluripotent stem cells (iPSCs) in gene therapy research is a powerful tool to study genetic diseases and to develop new therapies. iPSCs are adult cells that have been reprogrammed to an embryonic stem cell-like state, which allows them to differentiate into any cell type in the body. By reprogramming patient cells, scientists can create iPSCs that carry the genetic mutations responsible for a particular disease. These iPSCs can then be differentiated into the specific cell type affected by the disease, creating a laboratory model that closely mimics the patient’s condition.

Gene therapy is a method to correct a genetic disease by introducing a functional gene into the cells of an affected patient. iPSCs models allow for a preclinical screening of the gene therapy vectors, to test the efficiency, specificity and safety of the gene delivery system in the affected cell type. Additionally, iPSCs models can be used to study the long-term effects of gene therapy and to develop new strategies to improve the efficacy of gene therapy.

Overall, iPSCs models offer researchers a way to study genetic diseases in a controlled, laboratory setting, and to develop new therapies that can be tested in clinical trials.

Scaled in vitro platforms for gene therapy groups 

In one review, Capella et al. report on the potential of induced pluripotent stem cells to test gene therapy approaches for neuromuscular and motor neuron disorders.

They cite the ‘major advance that iPSCs offer in the the development of human disease models with the the concept of “disease in a dish,” using a generation of patient-specific models in vitro providing a new platform for gene therapy drug discovery.

Importantly, precision-medicine approaches to treat monogenic disorders exploit iPSCs potential for the selection and validation of lead candidates for conditions including Duchenne muscular dystrophy and 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.

The review describes the advantages for the use of this versatile tool for gene therapy, along with the challenges associated with the viral vector transduction of iPSCs.

Front. Cell Dev. Biol., 13 April 2021
Sec. Stem Cell Research
Volume 9 – 2021 | https://doi.org/10.3389/fcell.2021.662837