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CRISPR/Cas9-mediated correction of mutated copper transporter ATP7B
Wilson's disease (WD) is a monogenetic liver disease that is based on a mutation of the ATP7B gene and leads to a functional deterioration in copper (Cu) excretion in the liver. The excess Cu accumulates in various organs such as the liver and brain. WD patients show clinical heterogeneity, whi...
Autores principales: | Pöhler, Michael, Guttmann, Sarah, Nadzemova, Oksana, Lenders, Malte, Brand, Eva, Zibert, Andree, Schmidt, Hartmut H., Sandfort, Vanessa |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7526882/ https://www.ncbi.nlm.nih.gov/pubmed/32997714 http://dx.doi.org/10.1371/journal.pone.0239411 |
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