Abstract
Gene therapy holds the promise of correcting a genetic defect. It can be achieved with the introduction of a normal wild-type transgene into specific cells of the patient where the endogenous gene is underexpressing or by the introduction of a therapeutic agent, such as, antisense oligonucleotides (AON) or small interfering RNA (siRNA) to inhibit transcription and/or translation of an overexpressing endogenous gene or a cancer causing oncogene. Gene therapy has been utilized for vaccination and for the treatment of several diseases, such as, cancer, viral infections and dermatological diseases. However, there are many hurdles to overcome in developing effective gene-based therapeutics, including cellular barriers, enzymatic degradation and rapid clearance after administration. Successful transfer of nucleic acids (e.g. plasmid DNA, AON, siRNA, small hairpin RNA and micro RNA) into cells usually relies on the use of efficient carriers, commonly viral or non-viral vectors. Presently, viral vectors are more efficient than non-viral systems. However, immunogenicity, inflammatory reactions and problems associated with scale-up limit their clinical use. The ideal carriers for gene delivery should be safe and yet ensure that the DNA/RNA survives the extra- and intracellular environment and efficiently transfer to the appropriate cellular compartments. This review discusses some of the strategies that have been employed to overcome the barriers towards successful gene delivery.
Keywords: Gene therapy, nucleic acids delivery, viral vectors, non-viral vectors, plasmid DNA, antisense oligonucleotides, small interfering RNA, small hairpin RNA, micro RNA
Current Drug Delivery
Title: Non-Viral Nucleic Acid Delivery: Key Challenges and Future Directions
Volume: 8 Issue: 3
Author(s): Mahmoud Elsabahy, Adil Nazarali and Marianna Foldvari
Affiliation:
Keywords: Gene therapy, nucleic acids delivery, viral vectors, non-viral vectors, plasmid DNA, antisense oligonucleotides, small interfering RNA, small hairpin RNA, micro RNA
Abstract: Gene therapy holds the promise of correcting a genetic defect. It can be achieved with the introduction of a normal wild-type transgene into specific cells of the patient where the endogenous gene is underexpressing or by the introduction of a therapeutic agent, such as, antisense oligonucleotides (AON) or small interfering RNA (siRNA) to inhibit transcription and/or translation of an overexpressing endogenous gene or a cancer causing oncogene. Gene therapy has been utilized for vaccination and for the treatment of several diseases, such as, cancer, viral infections and dermatological diseases. However, there are many hurdles to overcome in developing effective gene-based therapeutics, including cellular barriers, enzymatic degradation and rapid clearance after administration. Successful transfer of nucleic acids (e.g. plasmid DNA, AON, siRNA, small hairpin RNA and micro RNA) into cells usually relies on the use of efficient carriers, commonly viral or non-viral vectors. Presently, viral vectors are more efficient than non-viral systems. However, immunogenicity, inflammatory reactions and problems associated with scale-up limit their clinical use. The ideal carriers for gene delivery should be safe and yet ensure that the DNA/RNA survives the extra- and intracellular environment and efficiently transfer to the appropriate cellular compartments. This review discusses some of the strategies that have been employed to overcome the barriers towards successful gene delivery.
Export Options
About this article
Cite this article as:
Elsabahy Mahmoud, Nazarali Adil and Foldvari Marianna, Non-Viral Nucleic Acid Delivery: Key Challenges and Future Directions, Current Drug Delivery 2011; 8 (3) . https://dx.doi.org/10.2174/156720111795256174
DOI https://dx.doi.org/10.2174/156720111795256174 |
Print ISSN 1567-2018 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5704 |
Call for Papers in Thematic Issues
Advances of natural products, bio-actives and novel drug delivery system against emerging viral infections
Due to the increasing prevalence of viral infections and the ability of these human pathogens to develop resistance to current treatment strategies, there is a great need to find and develop new compounds to combat them. These molecules must have low toxicity, specific activity and high bioavailability. The most suitable ...read more
Electrospun Fibers as Drug Delivery Systems
In recent years, electrospun fibers have attracted considerable attention as potential platforms for drug delivery due to their distinctive properties and adaptability. These fibers feature a notable surface area-to-volume ratio and can be intentionally designed with high porosity, facilitating an increased capacity for drug loading and rendering them suitable for ...read more
Emerging Nanotherapeutics for Mitigation of Neurodegenerative Disorders
Conditions affecting the central nervous system (CNS) present a significant hurdle due to limited access of both treatments and diagnostic tools for the brain. The blood-brain barrier (BBB) acts as a barrier, restricting the passage of molecules from the bloodstream into the brain. The most formidable challenge facing scientists is ...read more
Nanotechnology Based Chemotherapy for the treatment of Head & Neck Cancer
The escalating recurrence rates observed in Head and Neck cancer, particularly within the chemo-therapeutically treated cohort (50-60%), can be attributed to the non-selective nature of current anticancer drug delivery modalities. In this context, nanotechnology-based drug delivery systems emerge as a promising avenue for achieving precise localization of therapeutic agents to ...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
-
Anabolic Factors in Degenerative Joint Disease
Current Drug Targets 4-aryl/heteroaryl-4H-fused Pyrans as Anti-proliferative Agents: Design, Synthesis and Biological Evaluation
Anti-Cancer Agents in Medicinal Chemistry The Radio-Sensitizing Effect of Pharmacological Concentration of Ascorbic Acid on Human Pancreatic Cancer Cells
Anti-Cancer Agents in Medicinal Chemistry Prevalence of Dilated Cardiomyopathy in HIV-Infected African Patients Not Receiving HAART: A Multicenter, Observational, Prospective, Cohort Study in Rwanda
Current HIV Research Detection of Predictive Markers for Therapeutic Stratification of Salivary Glands Tumors
Current Drug Targets Antiangiogenic Therapy and Ovarian Cancer
Current Women`s Health Reviews Disordered Interactome of Human Papillomavirus
Current Pharmaceutical Design Prospects for Plant-Derived Chemopreventive Agents Exhibiting Multiple Mechanisms of Action
Current Medicinal Chemistry - Anti-Cancer Agents Journey to the Center of the Cell: Current Nanocarrier Design Strategies Targeting Biopharmaceuticals to the Cytoplasm and Nucleus
Current Pharmaceutical Design Targeting Microparticle Biogenesis: A Novel Approach to the Circumvention of Cancer Multidrug Resistance
Current Cancer Drug Targets Overview of Genomic Insights into Chicken Growth Traits Based on Genome- Wide Association Study and microRNA Regulation
Current Genomics Inhibitors of Lactate Dehydrogenase Isoforms and their Therapeutic Potentials
Current Medicinal Chemistry PET Imaging for Gene & Cell Therapy
Current Gene Therapy Leukotriene A4 Hydrolase as a Target for Cancer Prevention and Therapy
Current Cancer Drug Targets Editorial (Thematic Issue: Oncoviruses and Head and Neck Cancer: An Impending Facts)
Recent Patents on Biomarkers Inhibition of Highly Pathogenic Avian H5N1 Influenza Virus Replication by NanoRNA Oligonucleotides
Anti-Inflammatory & Anti-Allergy Agents in Medicinal Chemistry Engineered Biosynthesis of Medicinally Important Plant Natural Products in Microorganisms
Current Topics in Medicinal Chemistry Morpholino, siRNA, and S-DNA Compared: Impact of Structure and Mechanism of Action on Off-Target Effects and Sequence Specificity
Current Topics in Medicinal Chemistry New Therapeutic Applications of Phosphodiesterase 5 Inhibitors (PDE5-Is)
Current Medicinal Chemistry Bioactive Secondary Metabolites from Endophytic Fungi
Current Medicinal Chemistry