The identification of tissue-specific promoters for gene therapeutic constructs is one of the aims of complex tumor therapy. The genes encoding the fibroblast activation protein () and the connective tissue growth factor () can function in tumor-associated stromal cells but are practically inactive in normal adult cells. Accordingly, the promoters of these genes can be used to develop vectors targeted to the tumor microenvironment. However, the efficiency of these promoters within genetic constructs remains underexplored, particularly, at the organism level. Here, we used the model of embryos to study the efficiency of transient expression of marker genes under the control of promoters of the , , and immediate early genes of (CMV). Within 96 h after the injection of vectors, the and CMV promoters provided similar equal efficiency of reporter protein accumulation. In the case of the promoter, a high level of reporter protein accumulation was observed only in certain zebrafish individuals that were considered developmentally abnormal. Disturbed embryogenesis was the factor of changes in the exogenous promoter function. The data obtained make a significant contribution to understanding the function of the human and promoters within vectors to assess their potential in gene therapy.
Download full-text PDF |
Source |
---|---|
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10138699 | PMC |
http://dx.doi.org/10.3390/ijms24087192 | DOI Listing |
J Biotechnol
January 2025
Institute of Molecular Biotechnology (IMBT), BOKU University, Vienna, Austria.
Efficient recombinant protein production requires mammalian stable cell lines or often relies on inefficient transfection processes. Baculoviral transduction of mammalian cells (BacMam) offers cost-effective and robust gene transfer and straightforward scalability. The advantages over conventional approaches are, no need of high biosafety level laboratories, efficient transduction of various cell types and transfer of large transgenes into host cells.
View Article and Find Full Text PDFAlzheimers Dement
December 2024
Whitehead Institute, Cambridge, MA, USA.
Background: Alzheimer's disease (AD) and other neurodegenerative diseases (NDs) cause substantial health-related and economic burdens, but progress towards preventative or ameliorative treatments has been limited. Genome-wide association studies have identified hundreds of risk loci containing single nucleotide polymorphisms (SNPs) that alter risk for these diseases, but >90% of these SNPs are in noncoding regions, which are cell type-specific and difficult to study. To address this gap, we have characterized the epigenomes of iPSC-derived neuronal and glial cells and performed CRISPRi single cell screening to dissect the molecular and cellular mechanisms underlying 10 ND risk loci.
View Article and Find Full Text PDFJ Biotechnol
December 2024
Featured Laboratory for Biosynthesis and Target Discovery of Active Components of Traditional Chinese Medicine, School of Traditional Chinese Medicine, Binzhou Medical University, Yantai 264003, PR China. Electronic address:
Appl Environ Microbiol
December 2024
Department of Microbiology, Harvard Medical School, Boston, Massachusetts, USA.
Front Microbiol
December 2024
Department of Epidemiology, School of Public Health, Harbin Medical University, Harbin, China.
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!