Hepatocyte resident afflictions continue to affect the human population unabated. The
asialoglycoprotein receptor (ASGPR) is primarily expressed on hepatocytes and minimally …

The widespread clinical implementation of gene therapy requires the ability to stably
integrate genetic information through gene transfer vectors in a safe, effective, and …

Molecular medicine has entered a high-tech age that provides curative treatments of
complex genetic diseases through genetically engineered cellular medicinal products. Their …

Over the past decade, the Slee** Beauty (SB) transposon system has been developed as
the leading non-viral vector for gene therapy. This vector combines the advantages of …

Transposons derived from Slee** Beauty (SB), piggyBac (PB), or Tol2 typically require
cotransfection of transposon DNA with a transposase either as an expression plasmid or …

Abstract The Slee** Beauty transposon system is a nonviral DNA transfer tool capable of
efficiently mediating transposition‐based, stable integration of DNA sequences of choice …

Transposons have emerged as promising vectors for gene therapy that can potentially
overcome some of the limitations of commonly used viral vectors. Transposons stably …

Recent results confirm that long‐term expression of therapeutic transgenes can be achieved
by using a transposon‐based system in primary stem cells and in vivo. Transposable …

Slee** Beauty (SB) is the first synthetic DNA transposon that was shown to be active in a
wide variety of species. Here, we review studies from the last two decades addressing both …

Fabry disease (FD) results from a lack of activity of the lysosomal enzyme α-Galactosidase A
(α-Gal A), leading to the accumulation of glycosphingolipids in several different cell types …