The authors used CRISPR/Cas9 to correct β-Thal iPSCs, gene-corrected cells exhibit normal karyotypes and fully pluripotency as hES, showed no off-targeting effects. Their results shown that the gene-corrected β-Thal iPS cell lines restored HBB expression and reduced reactive oxygen species (ROS) production.
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The authors used CRISPR/Cas9 to correct β-Thal iPSCs, gene-corrected cells exhibit normal karyotypes and fully pluripotency as hES, showed no off-targeting effects. Their results shown that the gene-corrected β-Thal iPS cell lines restored HBB expression and reduced reactive oxygen species (ROS) production.
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