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 …

Recent advances in the development of genome editing technologies based on
programmable nucleases have substantially improved our ability to make precise changes …

Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to
ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by …

Base editing by nucleotide deaminases linked to programmable DNA-binding proteins
represents a promising approach to permanently remedy blood disorders, although its …

Immunodeficient mice engrafted with functional human cells and tissues, that is, humanized
mice, have become increasingly important as small, preclinical animal models for the study …

The recent development of CRISPR–Cas systems as easily accessible and programmable
tools for genome editing and regulation is spurring a revolution in biology. Paired with the …

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 …

Background CCR5 is the major coreceptor for human immunodeficiency virus (HIV). We
investigated whether site-specific modification of the gene (“gene editing”)—in this case, the …

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 …