@inbook{eee29e2c26364d78b6dd93bf93862bda,
title = "Manipulation of Proteostasis Networks in Transgenic ZAAT Zebrafish via CRISPR–Cas9 Gene Editing",
abstract = "The CRISPR–Cas9 genome editing system is used to induce mutations in genes of interest resulting in the loss of functional protein. A transgenic zebrafish α1-antitrypsin deficiency (AATD) model displays an unusual phenotype, in that it lacks the hepatic accumulation of the misfolding Z α1-antitrypsin (ZAAT) evident in human and mouse models. Here we describe the application of the CRISPR–Cas9 system to generate mutant zebrafish with defects in key proteostasis networks likely to be involved in the hepatic processing of ZAAT in this model. We describe the targeting of the atf6a and man1b1 genes as examples.",
keywords = "AATD, Atf6a, CRISPR–Cas9, Man1b1, Zebrafish, α1-antitrypsin",
author = "Connie Fung and Miles, {Lee B.} and Bryson-Richardson, {Robert J.} and Bird, {Phillip I.}",
note = "Funding Information: This work was funded by the Alpha-1 Foundation (USA). We thank the staff of the Monash FishCore facility for zebrafish care and maintenance. Publisher Copyright: {\textcopyright} The Author(s), under exclusive license to Springer Science+Business Media, LLC, part of Springer Nature 2024.",
year = "2024",
doi = "10.1007/978-1-0716-3605-3_3",
language = "English",
isbn = "9781071636046",
series = "Methods in Molecular Biology",
publisher = "Humana Press",
pages = "19--32",
editor = "{Bristow }, {Cynthia L}",
booktitle = "Alpha-1 Antitrypsin",
address = "United States of America",
}