Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors
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...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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Elsevier
2019-03-01
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| Series: | Molecular Therapy: Methods & Clinical Development |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050119300038 |
| _version_ | 1818671163758346240 |
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| author | Simone Merlin Antonia Follenzi |
| author_facet | Simone Merlin Antonia Follenzi |
| author_sort | Simone Merlin |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-17T07:19:38Z |
| format | Article |
| id | doaj.art-962b90333e1c4ddda9217b4352cfa648 |
| institution | Directory Open Access Journal |
| issn | 2329-0501 |
| language | English |
| last_indexed | 2024-12-17T07:19:38Z |
| publishDate | 2019-03-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Therapy: Methods & Clinical Development |
| spelling | doaj.art-962b90333e1c4ddda9217b4352cfa6482022-12-21T21:58:49ZengElsevierMolecular Therapy: Methods & Clinical Development2329-05012019-03-0112223232Transcriptional Targeting and MicroRNA Regulation of Lentiviral VectorsSimone Merlin0Antonia Follenzi1Department of Health Sciences, School of Medicine, University of Piemonte Orientale, Novara, ItalyDepartment of Health Sciences, School of Medicine, University of Piemonte Orientale, Novara, Italy; Corresponding author: Antonia Follenzi, MD, PhD, Department of Health Sciences, School of Medicine, University of Piemonte Orientale, via Gen. Paolo Solaroli, 17, 28100 Novara, Italy.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.http://www.sciencedirect.com/science/article/pii/S2329050119300038 |
| spellingShingle | Simone Merlin Antonia Follenzi Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors Molecular Therapy: Methods & Clinical Development |
| title | Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors |
| title_full | Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors |
| title_fullStr | Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors |
| title_full_unstemmed | Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors |
| title_short | Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors |
| title_sort | transcriptional targeting and microrna regulation of lentiviral vectors |
| url | http://www.sciencedirect.com/science/article/pii/S2329050119300038 |
| work_keys_str_mv | AT simonemerlin transcriptionaltargetingandmicrornaregulationoflentiviralvectors AT antoniafollenzi transcriptionaltargetingandmicrornaregulationoflentiviralvectors |