Genetically modified cells of haematopoietic and lymphocytic lineages could provide potentially curative treatments for a wide range of inherited and acquired diseases. However, this application is limited in mouse models by the low efficiency of lentiviral vectors. To facilitate the rapid production of high-titre helper-free retroviral vectors for enhanced gene delivery, multiple modifications...

OBJECTIVES In this study, we describe an efficient approach for stable knockdown of adenosine kinase (ADK) using lentiviral system, in an astrocytoma cell line and in human Wharton's jelly mesenchymal stem cells (hWJMSCs). These sources of stem cells besides having multilineage differentiation potential and immunomodulatory activities, are easily available in unlimited numbers, do not raise eth...

High-titer self-inactivating human immunodeficiency virus type-1 (HIV-1)-based vectors expressing the green fluorescent protein reporter gene that contained the central polypurine and termination tract and the woodchuck hepatitis virus posttranscriptional regulatory element were constructed. Transduction efficiency and biodistribution were determined, following systemic administration of these ...

The vascular wall (VW) serves as a niche for mesenchymal stem cells (MSCs). In general, tissue-specific stem cells differentiate mainly to the tissue type from which they derive, indicating that there is a certain code or priming within the cells as determined by the tissue of origin. Here we report the in vitro generation of VW-typical MSCs from induced pluripotent stem cells (iPSCs), based on...

Several diseases and injuries of the central nervous system could potentially be treated by delivery of an enzyme, which might most effectively be achieved by gene therapy. In particular, the bacterial enzyme chondroitinase ABC is beneficial in animal models of spinal cord injury. We have adapted the chondroitinase gene so that it can direct secretion of active chondroitinase from mammalian cel...

Development of a Pseudotyped Lentiviral Vector–Based Neutralization Assay for 1 Chikungunya Virus Infection 2 3 Running Title: CHIKV-pseudotyped lentiviral vector–based NT assay 4 5 Natsuko Kishishita, Naokazu Takeda, Atchareeya Anuegoonpipat, and Surapee 6 Anantapreecha 7 8 Thailand-Japan Research Collaboration Center on Emerging and Reemerging Infections 9 (RCC-ERI), Nonthaburi, Thailand 10 R...

Lentiviral vectors are used in laboratories around the world for in vivo and ex vivo delivery of gene therapies, and increasingly clinical investigation as well as preclinical applications. The third-generation lentiviral vector system has many advantages, including high packaging capacity, stable gene expression in both dividing and post-mitotic cells, and low immunogenicity in the recipient o...

Development of a Pseudotyped Lentiviral Vector–Based Neutralization Assay for 1 Chikungunya Virus Infection 2 3 Running Title: CHIKV-pseudotyped lentiviral vector–based NT assay 4 5 Natsuko Kishishita, Naokazu Takeda, Atchareeya Anuegoonpipat, and Surapee 6 Anantapreecha 7 8 Thailand-Japan Research Collaboration Center on Emerging and Reemerging Infections 9 (RCC-ERI), Nonthaburi, Thailand 10 R...

OBJECTIVES Lentiviral vectors have been used successfully to rapidly produce decigram quantities of active recombinant proteins in mammalian cell lines. To optimize the protein production platform, the roles of Ubiquitous Chromatin Opening Element (UCOE), an insulator, and selected promoters were evaluated based on efficiency and stability of foreign gene expression mediated by lentiviral vecto...

Gene transfer through retrograde axonal transport of viral vectors offers a substantial advantage for analyzing roles of specific neuronal pathways or cell types forming complex neural networks. This genetic approach may also be useful in gene therapy trials by enabling delivery of transgenes into a target brain region distant from the injection site of the vectors. Pseudotyping of a lentiviral...