Researchers at the University of Helsinki, University Hospital Helsinki have devised an approach to rapidly correct genetic defects in cultured cells of patients.
The procedure produces genetically corrected autologous stem cells from a 3 mm skin biopsy from patients with different genetic diseases. The stem cells that have been corrected are vital for research and development of new treatments.
The scientists based the new method on previous innovative research in the field of stem cells and gene editing, including two Nobel-prize-winning techniques. The Nobel Prize in 2012 was won for the invention of Induced Pluripotent Stem cells, iPSCs derived from differentiated cells. Another technique is the CRISPR-Cas9 “gene scissors” invention, which was awarded the award in 2020. This new method combines both these methods to correct gene mutations that can cause the inherited diseases, and generates fully functional stem cells.
New treatments for genetic diseases
The long-term goal of the researchers is to create autologous cells that can be used for therapeutic purposes. The cells of the patient could be used to circumvent immune-related obstacles that hinder transplantation of organs and tissues from donors. Sami Jalil, an Argentinian PhD student at the Biomedicum Helsinki Stem Cell Center who developed the new method, and it was published in the Stem Cell Reportsthe journal of The International Society of Stem Cell Research.
There are more than 6000 identified inheritable diseases, caused by different gene alterations. Some of them are being treated with a stem cell or organ transplant from a healthy donor, when there is one available.
“Our new method is more accurate and faster than previous methods for correcting the DNA errors. The speed of the process makes it more efficient and lessens the risk of unwanted changes,” says adjunct professor Kirmo Wartiovaara, who supervised the work.
In the ideal conditions, we have achieved up to 100 % efficacy, although one has to remember that the correction of cultured cells is far from the clinically proven applications. But it is a very positive start.”
Kirmo Wartiovaara
Jalil, S., et al. (2021) Simultaneous high-efficiency base editing and reprogramming patient fibroblasts. Stem Cell Reports. doi.org/10.1016/j.stemcr.2021.10.017.
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