CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I

CRISPR/Cas9 technology offers novel approaches for the development of new therapies for many unmet clinical needs, including a significant number of inherited monogenic diseases. However, in vivo correction of disease-causing genes is still inefficient, especially for those diseases without selectiv...

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Pubblicato in:Nat Commun
Autori principali: Zabaleta, Nerea, Barberia, Miren, Martin-Higueras, Cristina, Zapata-Linares, Natalia, Betancor, Isabel, Rodriguez, Saray, Martinez-Turrillas, Rebeca, Torella, Laura, Vales, Africa, Olagüe, Cristina, Vilas-Zornoza, Amaia, Castro-Labrador, Laura, Lara-Astiaso, David, Prosper, Felipe, Salido, Eduardo, Gonzalez-Aseguinolaza, Gloria, Rodriguez-Madoz, Juan R.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group UK 2018
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6303323/
https://ncbi.nlm.nih.gov/pubmed/30575740
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-018-07827-1
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