scholarly journals Non-replicating expression vectors: applications in vaccine development and gene therapy

1996 ◽  
Vol 116 (3) ◽  
pp. 241-256 ◽  
Author(s):  
K. J. Limbach ◽  
E. Paoletti

SUMMARYThis review presents experimental, preclinical and clinical data illustrating the multiple uses of recombinant non-replicating virus vectors in the fields of immunoprophylaxis and gene therapy.

Genes ◽  
2019 ◽  
Vol 10 (3) ◽  
pp. 189 ◽  
Author(s):  
Kenneth Lundstrom

RNA viruses have been subjected to substantial engineering efforts to support gene therapy applications and vaccine development. Typically, retroviruses, lentiviruses, alphaviruses, flaviviruses rhabdoviruses, measles viruses, Newcastle disease viruses, and picornaviruses have been employed as expression vectors for treatment of various diseases including different types of cancers, hemophilia, and infectious diseases. Moreover, vaccination with viral vectors has evaluated immunogenicity against infectious agents and protection against challenges with pathogenic organisms. Several preclinical studies in animal models have confirmed both immune responses and protection against lethal challenges. Similarly, administration of RNA viral vectors in animals implanted with tumor xenografts resulted in tumor regression and prolonged survival, and in some cases complete tumor clearance. Based on preclinical results, clinical trials have been conducted to establish the safety of RNA virus delivery. Moreover, stem cell-based lentiviral therapy provided life-long production of factor VIII potentially generating a cure for hemophilia A. Several clinical trials on cancer patients have generated anti-tumor activity, prolonged survival, and even progression-free survival.


Viruses ◽  
2021 ◽  
Vol 13 (5) ◽  
pp. 779
Author(s):  
Man Teng ◽  
Yongxiu Yao ◽  
Venugopal Nair ◽  
Jun Luo

In recent years, the CRISPR/Cas9-based gene-editing techniques have been well developed and applied widely in several aspects of research in the biological sciences, in many species, including humans, animals, plants, and even in viruses. Modification of the viral genome is crucial for revealing gene function, virus pathogenesis, gene therapy, genetic engineering, and vaccine development. Herein, we have provided a brief review of the different technologies for the modification of the viral genomes. Particularly, we have focused on the recently developed CRISPR/Cas9-based gene-editing system, detailing its origin, functional principles, and touching on its latest achievements in virology research and applications in vaccine development, especially in large DNA viruses of humans and animals. Future prospects of CRISPR/Cas9-based gene-editing technology in virology research, including the potential shortcomings, are also discussed.


2016 ◽  
Vol 82 (8) ◽  
pp. 2240-2246 ◽  
Author(s):  
Alex I. Kanno ◽  
Cibelly Goulart ◽  
Henrique K. Rofatto ◽  
Sergio C. Oliveira ◽  
Luciana C. C. Leite ◽  
...  

ABSTRACTThe expression of many antigens, stimulatory molecules, or even metabolic pathways in mycobacteria such asMycobacterium bovisBCG orM. smegmatiswas made possible through the development of shuttle vectors, and several recombinant vaccines have been constructed. However, gene expression in any of these systems relied mostly on the selection of natural promoters expected to provide the required level of expression by trial and error. To establish a systematic selection of promoters with a range of strengths, we generated a library of mutagenized promoters through error-prone PCR of the strong PL5promoter, originally from mycobacteriophage L5. These promoters were cloned upstream of the enhanced green fluorescent protein reporter gene, and recombinantM. smegmatisbacteria exhibiting a wide range of fluorescence levels were identified. A set of promoters was selected and identified as having high (pJK-F8), intermediate (pJK-B7, pJK-E6, pJK-D6), or low (pJK-C1) promoter strengths in bothM. smegmatisandM. bovisBCG. The sequencing of the promoter region demonstrated that it was extensively modified (6 to 11%) in all of the plasmids selected. To test the functionality of the system, two different expression vectors were demonstrated to allow corresponding expression levels of theSchistosoma mansoniantigen Sm29 in BCG. The approach used here can be used to adjust expression levels for synthetic and/or systems biology studies or for vaccine development to maximize the immune response.


2009 ◽  
Vol 20 (1) ◽  
pp. 63-71 ◽  
Author(s):  
Hitoshi Yokoyama ◽  
Katsumi Sasaki ◽  
Michael E. Franks ◽  
William F. Goins ◽  
James R. Goss ◽  
...  

Gene Therapy ◽  
2020 ◽  
pp. 77-92
Author(s):  
Jeffrey S. Bartlett ◽  
Richard J. Samulski

2021 ◽  
Vol 32 (19-20) ◽  
pp. 1096-1119
Author(s):  
Josephine Macdonald ◽  
Jennifer Marx ◽  
Hildegard Büning

2021 ◽  
Author(s):  
Moataz Dowaidar

Transcriptomics is a rapidly growing field that generates new data that may be used on its own or in combination with existing clinical data to widen and affect the future of healthcare. While the majority of current applications are limited to research, a growing number of studies suggest that transcriptomics has applications in diagnostics, genomics-driven trial design, and the creation of personalized medicines. Blood samples can be collected in general practice and submitted to a central lab for analysis and interpretation before being provided to the doctor, allowing for greater clinical acceptance of experimental hypotheses. The transcriptome's immense complexity has been revealed by transcriptomics, and we're just beginning to understand how this translates to function, disease, and therapeutic options.


2020 ◽  
Vol 21 (4) ◽  
pp. 255-272 ◽  
Author(s):  
Chengwen Li ◽  
R. Jude Samulski

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