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

Published 2017 View Full Article

Authors

Keywords

humanized liver mouse model, liver xenograft, liver regeneration, α-1 antitrypsin deficiency, nuclease-free genome editing, gene editing, AAV, miRNA, shRNA, RNAi, AAT, AATD, A1AT

Journal

MOLECULAR THERAPY

Volume 25, Issue 11, Pages 2477-2489

Publisher

Elsevier BV

Online

2017-09-25

DOI

10.1016/j.ymthe.2017.09.020

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