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
-
Chemosensitization by Antisense Oligonucleotides Targeting MDM2
Current Cancer Drug Targets Hypoxia and Inflammation in Prostate Cancer Progression. Cross-talk with Androgen and Estrogen Receptors and Cancer Stem Cells
Endocrine, Metabolic & Immune Disorders - Drug Targets Vascular Endothelial Growth Factor in Central Nervous System Injuries – A Vascular Growth Factor Getting Nervous?
Current Neurovascular Research Advances in Regulating Tumorigenicity and Metastasis of Cancer Through TrkB Signaling
Current Cancer Drug Targets Fluorescence Molecular Imaging of Small Animal Tumor Models
Current Molecular Medicine Tumor Escape from Immune Response: Mechanisms and Targets of Activity
Current Drug Targets <i>In Vivo</i> Tracking of Novel SPIO-Molday ION Rhodamine-B™-Labeled Human Bone Marrow-Derived Mesenchymal Stem Cells After Lentivirus- Mediated COX-2 Silencing: A Preliminary Study
Current Gene Therapy Cell Cycle and Cancer: The G1 Restriction Point and the G1 / S Transition
Current Genomics Protein Kinases as Drug Targets in Human and Animal Diseases
Current Enzyme Inhibition Modulators of Acetylcholinesterase Activity: From Alzheimer's Disease to Anti-Cancer Drugs
Current Medicinal Chemistry The Role of CXCR3 in Neurological Diseases
Current Neuropharmacology Signal Transducer and Activator of Transcription 3 Signaling Pathway: A Potential Target in Sarcoma Treatment
Current Enzyme Inhibition Evolution and Structure of API5 and Its Roles in Anti-Apoptosis
Protein & Peptide Letters Clients and Oncogenic Roles of Molecular Chaperone gp96/grp94
Current Topics in Medicinal Chemistry Patent Selections
Recent Patents on Anti-Cancer Drug Discovery Evolving Drug Delivery Strategies to Overcome the Blood Brain Barrier
Current Pharmaceutical Design Role of Alterations in the Apoptotic Machinery in Sensitivity of Cancer Cells to Treatment
Current Pharmaceutical Design Tumour Re-Differentiation Effect of Retinoic Acid: A Novel Therapeutic Approach for Advanced Thyroid Cancer
Current Pharmaceutical Design Brain Tumor Detection Using Machine Learning and Deep Learning: A Review
Current Medical Imaging Non-Covalent Proteasome Inhibitors
Current Pharmaceutical Design