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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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2023
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