The advent of genome editing has transformed the therapeutic landscape for several
debilitating diseases, and the clinical outlook for gene therapeutics has never been more …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

Over the last 60 years, more than a million patients received hematopoietic cell
transplantation. Having incorporated multiple changes in clinical practices, it remains a …

The piggyBac DNA transposon is used widely in genome engineering applications. Unlike
other transposons, its excision site can be precisely repaired without leaving footprints and it …

Allogeneic CD19-specific chimeric antigen receptor (CAR) T cells with inactivated donor T
cell receptor (TCR) expression can be used as an" off-the-shelf" therapeutic modality for …

Chimeric antigen receptor (CAR) technology and its application to regulatory T cells (Tregs)
has garnered interest among researchers in the field of cell and gene therapy. Merging the …

Abstract The Sleeping Beauty (SB) transposon system is an efficient non-viral gene transfer
tool in mammalian cells, but its broad use has been hampered by uncontrolled transposase …

Developing safe and efficient non-viral delivery systems remains a major challenge for in
vivo applications of gene therapy, especially in cystic fibrosis. Unlike conventional cationic …

Over a decade ago, a landmark study that reported derivation of induced Pluripotent Stem
Cells (iPSCs) by reprogramming fibroblasts has transformed stem cell research attracting …

With the genetic portraits of all major human malignancies now available, we next face the
challenge of characterizing the function of mutated genes, their downstream targets …