Nucleases that cleave unique genomic sequences in living cells can be used for targeted
gene editing and mutagenesis. Here we develop a strategy for generating such reagents …

A set of yeast strains based on Saccharomyces cerevisiae S288C in which commonly used
selectable marker genes are deleted by design based on the yeast genome sequence has …

Targeted genetic engineering of human pluripotent cells is a prerequisite for exploiting their
full potential. Such genetic manipulations can be achieved using site-specific nucleases. …

The toolbox of rat genetics currently lacks the ability to introduce site-directed, heritable
mutations into the genome to create knockout animals. By using engineered zinc-finger …

To the Editor: The recent description of highly active transcription activator-like effector
nucleases (TALENs) 1 prompted us to explore their utility for in vivo genetic engineering in the …

Zinc-finger nucleases (ZFNs) are powerful tools for producing gene knockouts (KOs) with
high efficiency. Whereas ZFN-mediated gene disruption has been demonstrated in laboratory …

Down’s syndrome is a common disorder with enormous medical and social costs, caused
by trisomy for chromosome 21. We tested the concept that gene imbalance across an extra …

L1 elements are ubiquitous human transposons that replicate via an RNA intermediate. We
have reconstituted the initial stages of L1 element transposition in vitro. The reaction requires …

Retrotransposons have shaped eukaryotic genomes for millions of years. To analyze the
consequences of human L1 retrotransposition, we developed a genetic system to recover many …

Sickle cell disease (SCD) is characterized by a single point mutation in the seventh codon of
the β-globin gene. Site-specific correction of the sickle mutation in hematopoietic stem cells …