By correcting disease-causing mutations in stem cells from patients with certain degenerative disorders, researchers could generate new disease models and treatments.
Juan Carlos Izpisúa Belmonte at the Salk Institute for Biological Studies in La Jolla, California, and his colleagues engineered an adenovirus that efficiently fixes mutated portions of the lamin A (LMNA) gene in stem cells created by reprogramming patients' cells. Corrected cells from patients with Hutchinson–Gilford progeria syndrome, an accelerated ageing disorder, produced normal connective tissue cells.
The same virus also repaired different LMNA mutations underlying a milder premature-ageing disease, atypical Werner syndrome. Moreover, it mended LMNA mutations in adult mesenchymal stem cells, a focus of current regenerative therapies.
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Gene repair tool for stem cells. Nature 474, 8 (2011). https://doi.org/10.1038/474008c