Publication: Molecular Therapy. 8(1): 108-117.
Published Date: July 2003
Examined several parameters of the Sleeping Beauty (SB) system to improve it as a potential non-viral vector for gene therapy.
Investigations centered on three features: the carrying capacity of the transposon for efﬁcient integration into chromosomes of HeLa cells, the effects of overexpression of the SB transposase gene on transposition rates, and improvements in the activity of SB transposase to increase insertion rates of transgenes into cellular chromosomes. Authors further reﬁned the SB transposase to incorporate several amino acid substitutions, the result of which led to an improved transposase called SB11 that appears to be a potential gene-transfer tool for human gene therapy.