Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset
neuromuscular disorder characterized by progressive muscle weakness and atrophy …

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

Duchenne muscular dystrophy (DMD) is an X-linked hereditary disease characterized by
progressive muscle wasting due to modifications in the DMD gene (exon deletions …

Engineering cells for adoptive therapy requires overcoming limitations in cell viability and, in
the efficiency of transgene delivery, the duration of transgene expression and the stability of …

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

DNA transposons are primitive genetic elements which have colonized living organisms
from plants to bacteria and mammals. Through evolution such parasitic elements have …

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

Sleeping 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 …

Members of the Tc1/marinersuperfamily are probably the most widespread DNA
transposons in nature (1). However, these elements appear to be transpositionally inactive …

We previously demonstrated that the characteristic sequence of matrix attachment regions
(MARs) allows transgenes to be maintained episomally in CHO cells. In the present study …