In vivo CRISPR-Cas9 inhibition of hepatic LDH as treatment of primary hyperoxaluria
Genome-editing strategies, especially CRISPR-Cas9 systems, have substantially increased the efficiency of innovative therapeutic approaches for monogenic diseases such as primary hyperoxalurias (PHs). We have previously demonstrated that inhibition of glycolate oxidase using CRISPR-Cas9 systems repr...
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| Autori principali: | , , , , , , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Elsevier
2022-06-01
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| Serie: | Molecular Therapy: Methods & Clinical Development |
| Soggetti: | |
| Accesso online: | http://www.sciencedirect.com/science/article/pii/S2329050122000365 |
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