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

The development of programmable nucleases to introduce defined alterations in genomic
sequences has been a powerful tool for precision medicine. While several nucleases such …

Advances in genome engineering in the last decade, particularly in the development of
programmable nucleases, have made it possible to edit the genomes of most cell types …

The clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9
(CRISPR/Cas9) system is a versatile and convenient genome-editing tool with prospects in …

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 …

Gene therapy represents a significant potential to revolutionize the field of hematology with
applications in correcting genetic mutations, generating cell lines and animal models, and …

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 …

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 …

Highlights•CRISPR/Cas9 overcomes limitations of earlier targeted nuclease-mediated
methods.•CRISPR/Cas9 allows for insertions/deletions of multiple alleles in a single …

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 …