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2025-12-02|

Correction Issued for Study on Hemophilia A Treatment Using CRISPR-Cas9 Nickase to Integrate Enhanced FVIII Gene into AAVS1 Locus

by GOAI
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A correction has been issued for the article titled “Long-term correction of hemophilia A via integration of a functionally enhanced FVIII gene into the AAVS1 locus by nickase in patient-derived iPSCs,” originally published in *Nature*. The correction addresses specific details related to the study, which focused on using advanced gene-editing techniques to treat hemophilia A.

The original research explored integrating an enhanced Factor VIII (FVIII) gene into the AAVS1 locus of patient-derived induced pluripotent stem cells (iPSCs). Researchers utilized a CRISPR-Cas9 nickase system to achieve precise genetic modifications. The study aimed to provide long-term therapeutic effects for individuals with hemophilia A, a genetic disorder characterized by insufficient blood clotting due to defective or missing FVIII protein. The corrected information clarifies aspects of the methodology and findings presented in the initial publication. Further details are available through the journal’s official website.

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Date: December 2, 2025

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