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In this study, Jalil and colleagues develop and test a method for simultaneous hiPSC
reprogramming and RNA-delivered adenine base editing. Starting from primary patient-derived
fibroblasts, this approach yields several on-target edited isogenic hiPSC lines, with
remarkable speed and editing efficiency and without compromising their genetic integrity
or their pluripotency quality. The gene editing corrects the cellular disease phenotype.
Researchers at the University of Helsinki and Helsinki University Hospital have developed a method to accurately and rapidly correct genetic alterations in cultured patient cells. The method produces genetically corrected autologous pluripotent stem cells from a 2-3 mm skin biopsy of patients with different genetic diseases. The scientists based the new method on two Nobel Prize-winning techniques. The first technique is the invention of induced pluripotent stem cells from differentiated cells, which won the Nobel Prize in 2012. The other technique is the CRISPR-Cas9 innovation that won the prize in 2020. The new method combines these techniques to correct genetic alterations that cause inherited diseases and at the same time create new, fully functional stem cells. Their new system is much faster and more precise than older methods for correcting DNA errors, and the speed makes it easier and also decreases the risk of unwanted changes.