Preclinical and clinical advances in transposon-based gene therapy

J Tipanee, YC Chai, T VandenDriessche… - Bioscience …, 2017 - portlandpress.com
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 …

[HTML][HTML] Transposons: moving forward from preclinical studies to clinical trials

K ChuahMarinee - Human gene therapy, 2017 - liebertpub.com
Transposons have emerged as promising vectors for gene therapy that can potentially
overcome some of the limitations of commonly used viral vectors. Transposons stably …

PiggyBac transposon-mediated gene transfer in human cells

MH Wilson, CJ Coates, AL George - Molecular therapy, 2007 - cell.com
Transposons are mobile genetic elements that can be used to integrate transgenes into host
cell genomes. The piggyBac transposon system has been used for transgenesis of insects …

Gene transfer into genomes of human cells by the sleeping beauty transposon system

AM Geurts, Y Yang, KJ Clark, G Liu, Z Cui, AJ Dupuy… - Molecular Therapy, 2003 - cell.com
Abstract The Sleeping Beauty (SB) transposon system, derived from teleost fish sequences,
is extremely effective at delivering DNA to vertebrate genomes, including those of humans …

Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors

Z Izsvák, MKL Chuah, T VandenDriessche, Z Ivics - Methods, 2009 - Elsevier
Transposable elements can be considered as natural, non-viral gene delivery vehicles
capable of efficient genomic insertion. The plasmid-based transposon system of Sleeping …

Sleeping Beauty transposon‐mediated gene therapy for prolonged expression

PB Hackett, SC Ekker, DA Largaespada, RS McIvor - Advances in genetics, 2005 - Elsevier
Abstract The Sleeping Beauty (SB) transposon system represents a new vector for non‐viral
gene transfer that melds advantages of viruses and other forms of naked DNA transfer. The …

Transposons for gene therapy!

Z Ivics, Z Izsvak - Current gene therapy, 2006 - ingentaconnect.com
Gene therapy is a promising strategy for the treatment of several inherited and acquired
human diseases. Several vector platforms exist for the delivery of therapeutic nucleic acids …

Gene insertion and long-term expression in lung mediated by the Sleeping Beauty transposon system

LR Belur, JL Frandsen, AJ Dupuy, DH Ingbar… - Molecular Therapy, 2003 - cell.com
Gene transfer to the lung could provide important new treatments for chronic and acquired
lung diseases such as cystic fibrosis, α1-antitrypsin deficiency, emphysema, and cancer …

Assembly of the Tc1 and mariner transposition initiation complexes depends on the origins of their transposase DNA binding domains

B Benjamin, B Yves, AG Corinne - Genetica, 2007 - Springer
In this review, we focus on the assembly of DNA/protein complexes that trigger transposition
in eukaryotic members of the IS630–Tc1–mariner (ITm) super-family, the Tc1-and mariner …

Transposon vectors for gene‐trap insertional mutagenesis in vertebrates

KJ Clark, AM Geurts, JB Bell, PB Hackett - genesis, 2004 - Wiley Online Library
The function of most vertebrate genes remains unknown or uncertain. Insertional
mutagenesis offers one approach to identify and understand the function of these genes …