RNA drugs and RNA targets for small molecules: principles, progress, and challenges

AM Yu, YH Choi, MJ Tu - Pharmacological reviews, 2020 - ASPET
RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …

Rabbit models for the study of human atherosclerosis: from pathophysiological mechanisms to translational medicine

J Fan, S Kitajima, T Watanabe, J Xu, J Zhang… - Pharmacology & …, 2015 - Elsevier
Laboratory animal models play an important role in the study of human diseases. Using
appropriate animals is critical not only for basic research but also for the development of …

Optimized Sleeping Beauty transposons rapidly generate stable transgenic cell lines

E Kowarz, D Löscher, R Marschalek - Biotechnology journal, 2015 - Wiley Online Library
Stable gene expression in mammalian cells is a prerequisite for many in vitro and in vivo
experiments. However, either the integration of plasmids into mammalian genomes or the …

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 …

[HTML][HTML] Ferret models of viral pathogenesis

T Enkirch, V Von Messling - Virology, 2015 - Elsevier
Emerging and well-known viral diseases remain one the most important global public health
threats. A better understanding of their pathogenesis and mechanisms of transmission …

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 …

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 …

Dielectrophoresis-assisted 3D nanoelectroporation for non-viral cell transfection in adoptive immunotherapy

L Chang, D Gallego-Perez, X Zhao, P Bertani, Z Yang… - Lab on a Chip, 2015 - pubs.rsc.org
Current transfection technologies lead to significant inter-clonal variations. Previously we
introduced a unique electrotransfection technology, Nanochannel-Electroporation (NEP) …

Germline transgenesis in pigs by cytoplasmic microinjection of Sleeping Beauty transposons

Z Ivics, W Garrels, L Mátés, TY Yau, S Bashir, V Zidek… - Nature protocols, 2014 - nature.com
The pig has emerged as an important large animal model in biomedical and pharmaceutical
research. We describe a protocol for high-efficiency germline transgenesis and sustained …