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Cell-based lab study suggests gene rescue in the most common form of LCA may provide a viable treatment strategy

Research, led by US scientists from the Wynn Institute for Vision Research, University of Iowa, have demonstrated that lentiviral delivery of a functioning gene for centrosomal protein may be capable of rescuing one of the most common forms of LCA. The pre-clinical experiments, conducted on derived murine and patient-specific photoreceptor precursor cells, successfully delivered a faithful copy of the CEP290 gene to overcome a defect in ciliogenesis. Following delivery, the cultured target cells, harbouring mutations in the CEP290 gene, were shown to express full-length replacement transcript to provide a level of functional rescue.