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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Detalles Bibliográficos
Autores principales: Martinez-Turrillas, Rebeca, Martin-Mallo, Angel, Rodriguez-Diaz, Saray, Zapata-Linares, Natalia, Rodriguez-Marquez, Paula, San Martin-Uriz, Patxi, Vilas-Zornoza, Amaia, Calleja-Cervantes, María E., Salido, Eduardo, Prosper, Felipe, Rodriguez-Madoz, Juan R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
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

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