Contemporary Transposon Tools: A Review and Guide through Mechanisms and Applications of Sleeping Beauty, piggyBac and Tol2 for Genome Engineering

N Sandoval-Villegas, W Nurieva, M Amberger… - International journal of …, 2021 - mdpi.com
Transposons are mobile genetic elements evolved to execute highly efficient integration of
their genes into the genomes of their host cells. These natural DNA transfer vehicles have …

Gene therapy with the sleeping beauty transposon system

P Kebriaei, Z Izsvák, SA Narayanavari, H Singh… - Trends in Genetics, 2017 - cell.com
The widespread clinical implementation of gene therapy requires the ability to stably
integrate genetic information through gene transfer vectors in a safe, effective, and …

Prediction of base editor off-targets by deep learning

C Zhang, Y Yang, T Qi, Y Zhang, L Hou, J Wei… - Nature …, 2023 - nature.com
Due to the tolerance of mismatches between gRNA and targeting sequence, base editors
frequently induce unwanted Cas9-dependent off-target mutations. Here, to develop models …

Current strategies employed in the manipulation of gene expression for clinical purposes

HC Tsai, V Pietrobon, M Peng, S Wang, L Zhao… - Journal of Translational …, 2022 - Springer
Abnormal gene expression level or expression of genes containing deleterious mutations
are two of the main determinants which lead to genetic disease. To obtain a therapeutic …

Engineered Treg cells: The heir to the throne of immunotherapy

A Stucchi, F Maspes, E Montee-Rodrigues… - Journal of …, 2023 - Elsevier
Recently, increased interest in the use of Tregs as adoptive cell therapy for the treatment of
autoimmune diseases and transplant rejection had led to several advances in the field …

Going non-viral: the Sleeping Beauty transposon system breaks on through to the clinical side

M Hudecek, Z Izsvák, S Johnen, M Renner… - Critical reviews in …, 2017 - Taylor & Francis
Molecular medicine has entered a high-tech age that provides curative treatments of
complex genetic diseases through genetically engineered cellular medicinal products. Their …

Latest advances for the sleeping beauty transposon system: 23 years of insomnia but prettier than ever: refinement and recent innovations of the sleeping beauty …

M Amberger, Z Ivics - Bioessays, 2020 - Wiley Online Library
Abstract The Sleeping Beauty transposon system is a nonviral DNA transfer tool capable of
efficiently mediating transposition‐based, stable integration of DNA sequences of choice …

RNA-guided retargeting of Sleeping Beauty transposition in human cells

A Kovač, C Miskey, M Menzel, E Grueso… - Elife, 2020 - elifesciences.org
An ideal tool for gene therapy would enable efficient gene integration at predetermined sites
in the human genome. Here we demonstrate biased genome-wide integration of the …

Synergistic effects of combined immunotherapy strategies in a model of multifocal hepatocellular carcinoma

MC Ochoa, S Sanchez-Gregorio, CE de Andrea… - Cell Reports …, 2023 - cell.com
Immune checkpoint-inhibitor combinations are the best therapeutic option for advanced
hepatocellular carcinoma (HCC) patients, but improvements in efficacy are needed to …

Transposon-based CAR T cells in acute leukemias: where are we going?

CF Magnani, S Tettamanti, G Alberti, I Pisani, A Biondi… - Cells, 2020 - mdpi.com
Chimeric Antigen Receptor (CAR) T-cell therapy has become a new therapeutic reality for
refractory and relapsed leukemia patients and is also emerging as a potential therapeutic …