The use of precise, rationally designed gene-editing nucleases allows for targeted genome
and transcriptome modification, and at present, four major classes of nucleases are being …

Genome editing technologies have advanced significantly over the past few years, providing
a fast and effective tool to precisely manipulate the genome at specific locations. The three …

The possibility of editing complex genomes in a targeted fashion has revolutionized basic
research as well as biomedical and biotechnological applications in the last 5 years. The …

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 …

Hematopoietic stem and progenitor cells (HSPCs) have great therapeutic potential because
of their ability to both self-renew and differentiate. It has been proposed that, given their …

The tremendous evolution of genome-editing tools in the last two decades has provided
innovative and effective approaches for gene therapy of congenital and acquired diseases …

The rapid advancement of genome-editing techniques holds much promise for the field of
human gene therapy. From bacteria to model organisms and human cells, genome editing …

Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

The ongoing advent of genome editing with programmable nucleases, including zinc‐finger
nuclease (ZFN), TAL effector nuclease (TALEN), and Clustered Regularly Interspaced Short …

Targeted genome editing with designer nucleases, such as zinc finger nucleases, TALE
nucleases, and CRISPR-Cas nucleases, has heralded a new era in gene therapy. Genetic …