ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

T Gaj, CA Gersbach, CF Barbas - Trends in biotechnology, 2013 - cell.com
Zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs)
comprise a powerful class of tools that are redefining the boundaries of biological research …

TALENs: a widely applicable technology for targeted genome editing

JK Joung, JD Sander - Nature reviews Molecular cell biology, 2013 - nature.com
Engineered nucleases enable the targeted alteration of nearly any gene in a wide range of
cell types and organisms. The newly-developed transcription activator-like effector …

GDF15 mediates the effects of metformin on body weight and energy balance

AP Coll, M Chen, P Taskar, D Rimmington, S Patel… - Nature, 2020 - nature.com
Metformin, the world's most prescribed anti-diabetic drug, is also effective in preventing type
2 diabetes in people at high risk,. More than 60% of this effect is attributable to the ability of …

A guide to genome engineering with programmable nucleases

H Kim, JS Kim - Nature Reviews Genetics, 2014 - nature.com
Programmable nucleases—including zinc-finger nucleases (ZFNs), transcription activator-
like effector nucleases (TALENs) and RNA-guided engineered nucleases (RGENs) derived …

Recent advances in 2D and 3D in vitro systems using primary hepatocytes, alternative hepatocyte sources and non-parenchymal liver cells and their use in …

P Godoy, NJ Hewitt, U Albrecht, ME Andersen… - Archives of …, 2013 - Springer
This review encompasses the most important advances in liver functions and hepatotoxicity
and analyzes which mechanisms can be studied in vitro. In a complex architecture of nested …

FLASH assembly of TALENs for high-throughput genome editing

D Reyon, SQ Tsai, C Khayter, JA Foden… - Nature …, 2012 - nature.com
Engineered transcription activator–like effector nucleases (TALENs) have shown promise as
facile and broadly applicable genome editing tools. However, no publicly available high …

[HTML][HTML] Building consensus on definition and nomenclature of hepatic, pancreatic, and biliary organoids

A Marsee, FJM Roos, MMA Verstegen, F Roos… - Cell stem cell, 2021 - cell.com
Hepatic, pancreatic, and biliary (HPB) organoids are powerful tools for studying
development, disease, and regeneration. As organoid research expands, the need for clear …

Genome-editing technologies for gene and cell therapy

ML Maeder, CA Gersbach - Molecular therapy, 2016 - cell.com
Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …

CRISPR/Cas9 for genome editing: progress, implications and challenges

F Zhang, Y Wen, X Guo - Human molecular genetics, 2014 - academic.oup.com
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
(Cas) protein 9 system provides a robust and multiplexable genome editing tool, enabling …

Alpha1-Antitrypsin Deficiency

P Strnad, NG McElvaney… - New England Journal of …, 2020 - Mass Medical Soc
Alpha1-Antitrypsin Deficiency AAT is a protease inhibitor targeting neutrophil elastase. It
prevents the destruction of tissue, particularly in the lung, from elastase activity. AAT …