Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9

Bhaskar Chanda Stem Cell Park et al. used CRISPR-Cas9 in patient iPSCs to correct two large inversions that are the most common underlying mutations for severe hemophilia, and they show that the correction is functional by rescuing lethality in hemophiliac mice using iPSC-derived endothelial cells.

from Cell Stem Cell
via Bhaskar Chanda UHN


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