Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency

Hepatocytes represent an important target for gene therapy and editing of single-gene disorders. In α-1 antitrypsin (AAT) deficiency, one missense mutation results in impaired secretion of AAT. In most patients, lung damage occurs due to a lack of AAT-mediated protection of lung elastin from neutrop...

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Detalles Bibliográficos
Autores principales: Borel, Florie, Tang, Qiushi, Gernoux, Gwladys, Greer, Cynthia, Wang, Ziqiong, Barzel, Adi, Kay, Mark A., Shultz, Leonard D., Greiner, Dale L., Flotte, Terence R., Brehm, Michael A., Mueller, Christian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5675605/
https://www.ncbi.nlm.nih.gov/pubmed/29032169
http://dx.doi.org/10.1016/j.ymthe.2017.09.020

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