CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I
Substrate reduction therapies (SRT) are a promising therapeutic approach for monogenic inherited metabolic diseases. Here the authors evaluate the therapeutic potential of an in vivo CRISPR/Cas9-mediated SRT to treat primary hyperoxaluria type I and demonstrate its safety and efficacy.
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| Main Authors: | , , , , , , , , , , , , , , , , |
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| Format: | article |
| Language: | EN |
| Published: |
Nature Portfolio
2018
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| Subjects: | |
| Online Access: | https://doaj.org/article/4b179c1567b1437ea464d69ab6bf5455 |
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