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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...
Autores principales: | , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8971349/ https://www.ncbi.nlm.nih.gov/pubmed/35402636 http://dx.doi.org/10.1016/j.omtm.2022.03.006 |