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Resolution of hepatic fibrosis after ZFN-mediated gene editing in the PiZ mouse model of human α1-antitrypsin deficiency
α1-antitrypsin deficiency is most commonly caused by a mutation in exon-7 of SERPINA1 (SA1-ATZ), resulting in hepatocellular accumulation of a misfolded variant (ATZ). Human SA1-ATZ-transgenic (PiZ) mice exhibit hepatocellular ATZ accumulation and liver fibrosis. We hypothesized that disrupting the...
Autores principales: | Li, Yanfeng, Guha, Chandan, Asp, Patrik, Wang, Xia, Tchaikovskya, Tatyana L., Kim, Kenneth, Mendel, Matthew, Cost, Gregory J., Perlmutter, David H., Roy-Chowdhury, Namita, Fox, Ira J., Conway, Anthony, Roy-Chowdhury, Jayanta |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Lippincott Williams & Wilkins
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9974076/ https://www.ncbi.nlm.nih.gov/pubmed/36848094 http://dx.doi.org/10.1097/HC9.0000000000000070 |
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