PAM-flexible Cas9-mediated base editing of a hemophilia B mutation in induced pluripotent stem cells
Abstract Background Base editing via CRISPR-Cas9 has garnered attention as a method for correcting disease-specific mutations without causing double-strand breaks, thereby avoiding large deletions and translocations in the host chromosome. However, its reliance on the protospacer adjacent motif (PAM...
Main Authors: | , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Portfolio
2023-04-01
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Series: | Communications Medicine |
Online Access: | https://doi.org/10.1038/s43856-023-00286-w |