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Edited stem cells offer hope of precision therapy for blindness

Findings raise the possibility of treating blinding eye diseases using a patient's own corrected cells as replacement tissue.

Using a new technology for repairing disease genes--the much-talked-about CRISPR/Cas9 gene editing--University of Iowa researchers working together with Columbia University Medical Center ophthalmologists have corrected a blindness-causing gene mutation in stem cells derived from a patient. The result offers hope that eye diseases might one day be treated by personalized, precision medicine in which patients' own cells are used to grow replacement tissue.