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 …

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 …

Multidimensional control of therapeutic human cell function with synthetic gene circuits

HS Li, DV Israni, KA Gagnon, KA Gan, MH Raymond… - Science, 2022 - science.org
Synthetic gene circuits that precisely control human cell function could expand the
capabilities of gene-and cell-based therapies. However, platforms for develo** circuits in …

[HTML][HTML] Origins of programmable nucleases for genome engineering

S Chandrasegaran, D Carroll - Journal of molecular biology, 2016 - Elsevier
Genome engineering with programmable nucleases depends on cellular responses to a
targeted double-strand break (DSB). The first truly targetable reagents were the zinc finger …

Targeting DNA double-strand breaks with TAL effector nucleases

M Christian, T Cermak, EL Doyle, C Schmidt, F Zhang… - Genetics, 2010 - academic.oup.com
Engineered nucleases that cleave specific DNA sequences in vivo are valuable reagents for
targeted mutagenesis. Here we report a new class of sequence-specific nucleases created …

Compact zinc finger base editors that edit mitochondrial or nuclear DNA in vitro and in vivo

JCW Willis, P Silva-Pinheiro, L Widdup… - Nature …, 2022 - nature.com
DddA-derived cytosine base editors (DdCBEs) use programmable DNA-binding TALE
repeat arrays, rather than CRISPR proteins, a split double-stranded DNA cytidine …

C2H2 zinc finger proteins greatly expand the human regulatory lexicon

HS Najafabadi, S Mnaimneh, FW Schmitges… - Nature …, 2015 - nature.com
Abstract Cys2-His2 zinc finger (C2H2-ZF) proteins represent the largest class of putative
human transcription factors. However, for most C2H2-ZF proteins it is unknown whether they …

Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA)

JD Sander, EJ Dahlborg, MJ Goodwin, L Cade… - Nature …, 2011 - nature.com
Engineered zinc-finger nucleases (ZFNs) enable targeted genome modification. Here we
describe context-dependent assembly (CoDA), a platform for engineering ZFNs using only …

Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases

M Bibikova, M Golic, KG Golic, D Carroll - Genetics, 2002 - academic.oup.com
Zinc-finger nucleases (ZFNs) are hybrids between a nonspecific DNA-cleavage domain and
a DNA-binding domain composed of Cys2His2 zinc fingers. Because zinc fingers can be …

The discovery of zinc fingers and their applications in gene regulation and genome manipulation

A Klug - Annual review of biochemistry, 2010 - annualreviews.org
An account is given of the discovery of the classical Cys2His2 zinc finger, arising from the
interpretation of biochemical studies on the interaction of the Xenopus protein transcription …