New CRISPR technology helps revolutionize gene therapy

Written by Sharon Salt

Researchers from the National Institutes of Health (MD, USA) and Harvard University (MA, USA) have used second-generation CRISPR gene-editing technology termed ‘base editing’ to revolutionize gene therapy in order to tackle a genetic disorder called Hutchinson–Gilford progeria syndrome (HGPS). Their results, which have been tested in cultured fibroblasts and mice, were published in Nature. HGPS is typically caused by a dominant-negative C–G to T–A mutation in the LMNA gene. This mutation results in the production of a toxic protein known as progerin, which induces rapid aging and shortens the lifespan of children with progeria to approximately 14 years. Within the...

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