Korean iPSC derived cell provider, Nexel, presents a new model Nexel succeeding in making Wilson's disease model for screening new drugs using stem cells
Dec 17, 2020, 03:05
A Korean iPSC derived cell provider, Nexel, announced that Nexel has succeeded in creating a model that can evaluate the efficacy of a candidate substance for treating Wilson's disease using human pluripotent stem cells. The research results were published in the international journal 'Cells'.
Nexel is the first provider in Korea to successfully develop and commercialize functional somatic cells since it acquired the patent license for induced pluripotent stem cells from Kyoto University in Japan. Nexel said that this achievement is meaningful because it is the world's first Wilson’s disease model made without stem cells derived from the patient's somatic cells.
'Wilson's disease' is a congenital disorder of copper metabolism caused by a defect in the gene that produces the copper transport protein. Also it is a genetic disease in which excessive amounts of copper are accumulated mainly in the liver and brain basal ganglia. Liver disease mainly appears before age 15, and after age 15, it becomes a genetic disease that shows neurological symptoms, and the liver is the primary target disease. It is with a high prevalence rate of 1 in 30,000 people.
Conventional research on therapeutic agents for genetic diseases has generally made use of induced pluripotent stem cells derived from somatic cells of patients with the corresponding genetic diseases to create models for the development of therapeutic agents. However, due to the rarity of the disease, the genetic disease had a fundamental limitation in making it difficult to obtain the patient's somatic cells.
In order to overcome this limitation, Nexel presented a new model using pluripotent stem cells rather than the conventional method using somatic stem cells.
Nexel transformed normal stem cells into stem cells with a Wilson’s disease genetic mutation using CRISPR gene scissors, and then differentiated them into stem cells with a Wilson’s disease genetic mutation.
An official from Nexel said, "It is evaluated that Nexel has presented an alternative that can overcome the fundamental limitations of the production of existing genetic disease models including Wilson's disease. It is expected that it will be a way to help a lot.”
In particular, Nexel presented the possibility of developing cell therapy products in the future as it is possible to mass-produce patient-specific hepatocytes derived from iPS cells.
The CEO of Nexel said, "The success of this Wilson's disease model development has provided an opportunity to expand the business area through screening for genetic disease treatment and new drug development." Based on this achievement, it is being applied to model production."
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Written by Hanmir PRNews
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