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Cell Biology

3 published articlesPart of Cells & Molecular Biology

Latest in Cell Biology

Cells & Molecular BiologyCell BiologyPeer-reviewed

Gene-edited stem cells linked to multi-organ gains in FRDA mice

A single infusion of edited blood-forming stem cells was associated with donor-cell engraftment and better movement, cerebellar, heart and pancreatic measures in humanized Friedreich’s ataxia mice. The findings were incomplete and remain preclinical, with some abnormalities persisting and the proposed editing threshold not prospectively validated.

The Journal of clinical investigation4 min read