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Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors
- Source :
- Molecular Therapy. Methods & Clinical Development, Molecular Therapy: Methods & Clinical Development, Vol 12, Iss, Pp 223-232 (2019)
- Publication Year :
- 2019
- Publisher :
- American Society of Gene & Cell Therapy, 2019.
-
Abstract
- Gene expression regulation is the result of complex interactions between transcriptional and post-transcriptional controls, resulting in cell-type-specific gene expression patterns that are determined by the developmental and differentiation stage of pathophysiological conditions. Understanding the complexity of gene expression regulatory networks is fundamental to gene therapy, an approach which has the potential to treat and cure inherited disorders by delivering the correct gene to patient specific cells or tissues by means of both viral and non-viral vectors. Besides the issues of biosafety, in recent years efforts have focused on achieving a robust and sustained transgene expression, which attains a phenotypic correction in several diseases, while avoiding transgene-related adverse effects, such as overexpression-associated cytotoxicity and/or immune responses to the transgene. In this sense, the use of cell-type-specific promoters and microRNA target sequences (miRTs) in gene transfer expression cassettes have allowed for a restricted expression after gene transfer in several studies. This review will focus on the use of transcriptional and post-transcriptional regulation to achieve a highly specific and safe transgene expression, as well as their application in ex vivo and in vivo gene therapeutic approaches.
- Subjects :
- 0301 basic medicine
Regulation of gene expression
lcsh:QH426-470
lcsh:Cytology
Transgene
Genetic enhancement
Promoter
Computational biology
Biology
Phenotype
Article
lcsh:Genetics
03 medical and health sciences
030104 developmental biology
0302 clinical medicine
030220 oncology & carcinogenesis
Gene expression
microRNA
Genetics
Molecular Medicine
lcsh:QH573-671
Molecular Biology
Gene
Subjects
Details
- Language :
- English
- ISSN :
- 23290501
- Volume :
- 12
- Database :
- OpenAIRE
- Journal :
- Molecular Therapy. Methods & Clinical Development
- Accession number :
- edsair.doi.dedup.....06fc309ab0a0fdf96f7bd7a0232b36eb