Stem Cell Rep: New stem cell genetic modification technology or accelerated development of individualized therapy

Stem Cell Rep: New stem cell genetic modification technology or accelerated development of individualized therapy

November 19, 2015 Source: Bio Valley

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November 18, 2015 / Bio Valley BIOON / -- Recently, a research paper published in the international magazine Stem Cell Reports , scientists from the Murdoch Children's Research Institute (MCRI) in Melbourne designed through research A method that dramatically shortens the critical time for stem cell reprogramming and genetic modification, and related research or hope for the development of novel targeted therapies for the treatment of disease.

In the article, the researchers revealed how hereditary repaired stem cells are derived from the skin cells of patients in just two weeks. The conventional methods used in the past can take more than three months. Researcher Dr. Sara Howden said that in this study we combined two necessary steps to repair cells for potential therapy, first reprogramming adult cells into embryonic-like cells so that they can differentiate into target cells; These cells need to undergo a complex genetic editing process to correct the mutations caused by the disease.

The researchers successfully combined the above two steps to conduct research using skin cells from adult patients with retinal degeneration, which allows patients to receive genetically modified cells at the right time to accelerate the success of transplant therapy. Another researcher, Thomson, said that if you want to use the patient's specific iPS cells to guide the treatment, then a longer timeline may make the process very difficult; using the new method of this article we can use Cas9/CRISPR The technology successfully combines reprogramming and genetic modification to greatly reduce the time required.

Induced pluripotent stem cells have great potential in medical research because they can be derived from any organism in nature and can be differentiated into 220 different types of human body cells. At the same time, effective and precise modification of DNA in cells may help develop Individualized stem cell therapy to help treat patients with multiple genetic disorders. The next step in the research is to study blood samples, not only because blood samples are less invasive than skin biopsy, but also because they can effectively reduce the time to acquire repaired iPS cells.

In the end, the researchers said that this reprogramming and repair acceleration process may be very important in some cases. For example, patients with severe comprehensive immunodeficiency often die within a few years. However, scientists still need to obtain more before the treatment takes effect. Blood cells from stem cells can be studied. (Bio Valley Bioon.com)

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