[HTML][HTML] 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 …

[HTML][HTML] 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 …

[HTML][HTML] Noncoding RNA NORAD regulates genomic stability by sequestering PUMILIO proteins

S Lee, F Kopp, TC Chang, A Sataluri, B Chen… - Cell, 2016 - cell.com
Long noncoding RNAs (lncRNAs) have emerged as regulators of diverse biological
processes. Here, we describe the initial functional analysis of a poorly characterized human …

[HTML][HTML] TALE and TALEN genome editing technologies

S Becker, J Boch - Gene and Genome Editing, 2021 - Elsevier
TALEN were the first easy-to-use genome editing technology and sparked the genome
editing revolution. Their application in multiple species brought targeted mutagenesis to the …

Efficient genome editing in zebrafish using a CRISPR-Cas system

WY Hwang, Y Fu, D Reyon, ML Maeder, SQ Tsai… - Nature …, 2013 - nature.com
In bacteria, foreign nucleic acids are silenced by clustered, regularly interspaced, short
palindromic repeats (CRISPR)–CRISPR-associated (Cas) systems. Bacterial type II CRISPR …

High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

V Pattanayak, S Lin, JP Guilinger, E Ma… - Nature …, 2013 - nature.com
The RNA-programmable Cas9 endonuclease cleaves double-stranded DNA at sites
complementary to a 20-base-pair guide RNA. The Cas9 system has been used to modify …

Genome-editing technologies: principles and applications

T Gaj, SJ Sirk, S Shui, J Liu - Cold Spring Harbor …, 2016 - cshperspectives.cshlp.org
Targeted nucleases have provided researchers with the ability to manipulate virtually any
genomic sequence, enabling the facile creation of isogenic cell lines and animal models for …

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 …

[HTML][HTML] Genome editing with RNA-guided Cas9 nuclease in zebrafish embryos

N Chang, C Sun, L Gao, D Zhu, X Xu, X Zhu, JW Xiong… - Cell research, 2013 - nature.com
Recent advances with the type II clustered regularly interspaced short palindromic repeats
(CRISPR) system promise an improved approach to genome editing. However, the …

High-efficiency TALEN-based gene editing produces disease-resistant rice

T Li, B Liu, MH Spalding, DP Weeks, B Yang - Nature biotechnology, 2012 - nature.com
Figure 1 High-efficiency targeted gene editing using TALENs.(a) Overlapping elements
targeted by two pairs (1 and 2) of designer TALENs in the Os11N3 promoter.(b–d) …