Researchers at the University of Helsinki and University Hospital Helsinki have developed a method to precisely and rapidly correct genetic alterations in the cultured patient cells. The method, which combines base editing and reprogramming, produces genetically corrected autologous pluripotent stem cells from a 2–3 mm skin biopsy from patients with different genetic diseases.
The team’s long-term goal is to produce autologous cells with therapeutic properties. The use of the patient’s own corrected cells could help in avoiding the immunological challenges hampering the organ and tissue transplantation from a donor.
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“Our new system is much faster and more precise than the older methods in correcting the DNA errors, and the speed makes it easier and diminishes also the risk of unwanted changes,” says Kirmo Wartiovaara, senior author on the study published recently in Stem Cell Reports.
“In perfect conditions, we have reached up to 100 % efficacy, although one has to remember that the correction of cultured cells is still far away from proven therapeutic applications. But it is a very positive start” Wartiovaara adds.