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Correction of ring chromosomes in patient-derived induced pluripotent stem (iPS) cells

Reprogramming patient fibroblasts into iPS cells could help correct ring chromosome abnormalities for gene therapy applications. In fibroblasts from a patient with Miller-Dieker syndrome with a heterozygous ring chromosome 17, 4 of 6 iPS cell clones generated from the fibroblasts lost the ring chromosome and gained a second, identical copy of the other chromosome 17 because of a compensatory uniparental disomy mechanism. Clones that retained the ring chromosome were not viable through multiple passages. The findings were replicated in cells from two additional patients with chromosome 13 rings. Next steps include testing whether the strategy can be applied to other types of chromosomal abnormalities.

SciBX 7(5); doi:10.1038/scibx.2014.155
Published online Feb. 6, 2014

Unpatented; unavailable for licensing

Bershteyn, M. et al. Nature; published online Jan. 12, 2014;
Contact: Anthony Wynshaw-Boris, Case Western Reserve University, Cleveland, Ohio

Contact: Shinya Yamanaka, Gladstone Institute of Cardiovascular Disease, San Francisco, Calif.