Abstract
Gene therapy, the expression in cells of genetic material that has therapeutic activity, holds great promise for the treatment of a number of human diseases. A gene delivery vehicle, or vector, that may be of viral or non-viral origin, is generally used to carry the genetic material. Viral vectors have been developed that exclude immunogenic genes while taking advantage of the genes responsible for proficient integration of the viral genome into that of the host. In this way, viral vectors improve the probability of long-term expression of the therapeutic gene, whereas non-viral vectors, that are not as efficient at introducing and maintaining foreign gene expression, have the advantage of being non-pathogenic and non-immunogenic. Although thousands of patients have been involved in clinical trials for gene therapy, using hundreds of different protocols, true success has been limited. A major limitation of gene therapy approaches, especially when non-viral vectors are used, is the poor efficiency of DNA delivery to the nucleus; a crucial step to ensure ultimate expression of the therapeutic gene product. Here we review existing gene delivery approaches and, in particular, explore the possibility of enhancing non-viral gene delivery to the nucleus by incorporating specific nuclear targeting sequences in vectors, using a range of different strategies.
Current Drug Targets
Title: Gene Therapy: Optimising DNA Delivery to the Nucleus
Volume: 2 Issue: 4
Author(s): Melanie Johnson-Saliba and David A. Jans
Affiliation:
Abstract: Gene therapy, the expression in cells of genetic material that has therapeutic activity, holds great promise for the treatment of a number of human diseases. A gene delivery vehicle, or vector, that may be of viral or non-viral origin, is generally used to carry the genetic material. Viral vectors have been developed that exclude immunogenic genes while taking advantage of the genes responsible for proficient integration of the viral genome into that of the host. In this way, viral vectors improve the probability of long-term expression of the therapeutic gene, whereas non-viral vectors, that are not as efficient at introducing and maintaining foreign gene expression, have the advantage of being non-pathogenic and non-immunogenic. Although thousands of patients have been involved in clinical trials for gene therapy, using hundreds of different protocols, true success has been limited. A major limitation of gene therapy approaches, especially when non-viral vectors are used, is the poor efficiency of DNA delivery to the nucleus; a crucial step to ensure ultimate expression of the therapeutic gene product. Here we review existing gene delivery approaches and, in particular, explore the possibility of enhancing non-viral gene delivery to the nucleus by incorporating specific nuclear targeting sequences in vectors, using a range of different strategies.
Export Options
About this article
Cite this article as:
Johnson-Saliba Melanie and Jans A. David, Gene Therapy: Optimising DNA Delivery to the Nucleus, Current Drug Targets 2001; 2 (4) . https://dx.doi.org/10.2174/1389450013348245
DOI https://dx.doi.org/10.2174/1389450013348245 |
Print ISSN 1389-4501 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-5592 |
Call for Papers in Thematic Issues
New drug therapy for eye diseases
Eyesight is one of the most critical senses, accounting for over 80% of our perceptions. Our quality of life might be significantly affected by eye disease, including glaucoma, diabetic retinopathy, dry eye, etc. Although the development of microinvasive ocular surgery reduces surgical complications and improves overall outcomes, medication therapy is ...read more
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
Related Articles
-
Sodium Selenite Decreased HDAC Activity, Cell Proliferation and Induced Apoptosis in Three Human Glioblastoma Cells
Anti-Cancer Agents in Medicinal Chemistry Recent Advances in Antabuse (Disulfiram): The Importance of its Metal-binding Ability to its Anticancer Activity
Current Medicinal Chemistry Inhibitors of the TGF-β Superfamily and their Clinical Applications
Mini-Reviews in Medicinal Chemistry Meet Our Editorial Board Member
Current Drug Delivery Edema-mass Ratio Based On Magnetic Resonance Imaging As A Preoperative Diagnostic Factor For Posterior Fossa Metastasis
Current Medical Imaging Editorial (Thematic Issue: Emerging Concepts and Therapeutics Strategies for the Treatment of Brain Tumors)
Anti-Cancer Agents in Medicinal Chemistry Metabolomics and the Diagnosis of Human Diseases -A Guide to the Markers and Pathophysiological Pathways Affected
Current Medicinal Chemistry Activatable Optical Probes for the Detection of Enzymes
Current Organic Synthesis Targeting Ovarian Cancer-Initiating Cells
Anti-Cancer Agents in Medicinal Chemistry Review of Selected Patents for Cancer Therapy Targeting Tumor Angiogenesis
Recent Patents on Anti-Cancer Drug Discovery Polymeric Nanocarriers and Nanoreactors: A Survey of Possible Therapeutic Applications
Current Pharmaceutical Design Targeting Poly (ADP) Ribose Polymerase I (PARP-1) and PARP-1 Interacting Proteins for Cancer Treatment
Anti-Cancer Agents in Medicinal Chemistry Nano-pharmaceutical Formulations for Targeted Drug Delivery against HER2 in Breast Cancer
Current Cancer Drug Targets Peptide Self-Assemblies for Drug Delivery
Current Topics in Medicinal Chemistry Mechanisms of Tumor Cell Necrosis
Current Pharmaceutical Design Dietary and Plant Polyphenols Exert Neuroprotective Effects and Improve Cognitive Function in Cerebral Ischemia
Recent Patents on Food, Nutrition & Agriculture The Anti-Cancer Activity of Noscapine: A Review
Recent Patents on Anti-Cancer Drug Discovery The Interaction of NK Cells and Dendritic Cells in the Tumor Environment: How to Enforce NK Cell & DC Action Under Immunosuppressive Conditions?
Current Medicinal Chemistry Nutlins and Ionizing Radiation in Cancer Therapy
Current Pharmaceutical Design MicroRNA Targeting as a Therapeutic Strategy Against Glioma
Current Molecular Medicine