CRISPR RNA-guided FokI nucleases repair a PAH variant in a phenylketonuria model
The CRISPR/Cas9 system is a recently developed genome editing technique. In this study, we used a modified CRISPR system, which employs the fusion of inactive Cas9 (dCas9) and the FokI endonuclease (FokI-dCas9) to correct the most common variant (allele frequency 21.4%) in the phenylalanine hydroxyl...
| Main Authors: | , , , , , , |
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| Format: | Online |
| Language: | English |
| Published: |
Nature Publishing Group
2016
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5081532/ |