Programmable nucleases—including zinc-finger nucleases (ZFNs), transcription activator-
like effector nucleases (TALENs) and RNA-guided engineered nucleases (RGENs) derived …

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 …

We compared the ability of short hairpin RNA (shRNA) and CRISPR/Cas9 screens to identify
essential genes in the human chronic myelogenous leukemia cell line K562. We found that …

CRISPR-Cas9 screens are powerful tools for high-throughput interrogation of genome
function, but can be confounded by nuclease-induced toxicity at both on-and off-target sites …

Directly modulating the activity of genome-editing proteins has the potential to increase their
specificity by reducing activity following target locus modification. We developed Cas9 …

Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an
effective therapy for monogenic diseases of the blood and immune system. Here we …

Genome-editing technologies based on programmable nucleases have significantly
broadened our ability to make precise and direct changes in the genomic DNA of various …

The importance of safer approaches for gene therapy has been underscored by a series of
severe adverse events (SAEs) observed in patients involved in clinical trials for Severe …

The ability to regulate any protein of interest in living systems with small molecules remains
a challenge. We hypothesized that appending a hydrophobic moiety to the surface of a …

Zinc-finger nucleases (ZFNs) are versatile reagents that have redefined genome
engineering. Realizing the full potential of this technology requires the development of safe …