Gene Editing Seen to Restore Frataxin Level in Cells From Patients

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ADVANCE IN FREIDRICH’S ATAXIA RESEARCH: The CRISPR-Cas9 gene editing technique safely removed the gene expansion that causes Friedrich’s ataxia (FA), allowing for normal frataxin levels and more functional mitochondria in cells taken from patients, an early study reported. This work, and further experiments in mice, supports the potential of a stem cell approach to […]