Progress and prospects: zinc-finger nucleases as gene therapy agents

D Carroll - Gene therapy, 2008 - nature.com
Zinc-finger nucleases (ZFNs) are powerful tools for experimental gene manipulation. A
number of recent papers have shown how this technology can be applied effectively to …

Quantifying on-and off-target genome editing

A Hendel, EJ Fine, G Bao, MH Porteus - Trends in biotechnology, 2015 - cell.com
Genome editing with engineered nucleases is a rapidly growing field thanks to
transformative technologies that allow researchers to precisely alter genomes for numerous …

High-frequency modification of plant genes using engineered zinc-finger nucleases

JA Townsend, DA Wright, RJ Winfrey, F Fu, ML Maeder… - Nature, 2009 - nature.com
An efficient method for making directed DNA sequence modifications to plant genes (gene
targeting) is at present lacking, thereby frustrating efforts to dissect plant gene function and …

Rapid “open-source” engineering of customized zinc-finger nucleases for highly efficient gene modification

ML Maeder, S Thibodeau-Beganny, A Osiak, DA Wright… - Molecular cell, 2008 - cell.com
Custom-made zinc-finger nucleases (ZFNs) can induce targeted genome modifications with
high efficiency in cell types including Drosophila, C. elegans, plants, and humans. A …

Obligate ligation-gated recombination (ObLiGaRe): custom-designed nuclease-mediated targeted integration through nonhomologous end joining

M Maresca, VG Lin, N Guo, Y Yang - Genome research, 2013 - genome.cshlp.org
Custom-designed nucleases (CDNs) greatly facilitate genetic engineering by generating a
targeted DNA double-strand break (DSB) in the genome. Once a DSB is created, specific …

Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells

J Zou, ML Maeder, P Mali, SM Pruett-Miller… - Cell stem cell, 2009 - cell.com
We report here homologous recombination (HR)-mediated gene targeting of two different
genes in human iPS cells (hiPSCs) and human ES cells (hESCs). HR-mediated correction of …

TALEN-based gene correction for epidermolysis bullosa

MJ Osborn, CG Starker, AN McElroy, BR Webber… - Molecular Therapy, 2013 - cell.com
Recessive dystrophic epidermolysis bullosa (RDEB) is characterized by a functional deficit
of type VII collagen protein due to gene defects in the type VII collagen gene (COL7A1) …

Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs

P Xu, Y Tong, X Liu, T Wang, L Cheng, B Wang, X Lv… - Scientific reports, 2015 - nature.com
Abstract β-Thalassemia is one of the most common genetic blood diseases and is caused by
either point mutations or deletions in the β-globin (HBB) gene. The generation of patient …

Chromosomal translocations induced at specified loci in human stem cells

E Brunet, D Simsek, M Tomishima… - Proceedings of the …, 2009 - National Acad Sciences
The precise genetic manipulation of stem and precursor cells offers extraordinary potential
for the analysis, prevention, and treatment of human malignancies. Chromosomal …

Oligomerized pool engineering (OPEN): an'open-source'protocol for making customized zinc-finger arrays

ML Maeder, S Thibodeau-Beganny, JD Sander… - Nature protocols, 2009 - nature.com
Engineered zinc-finger nucleases (ZFNs) form the basis of a broadly applicable method for
targeted, efficient modification of eukaryotic genomes. In recent work, we described OPEN …