Non-Viral Delivery Systems in Gene Therapy and Vaccine Development
نویسندگان
چکیده
Gene therapy is the process of treating a particular disease through the introduction of genetic material in order to elicit a therapeutic benefit [Stone, 2010]. The defective gene of a diagnosed patient can be corrected by a number of different strategies such as “gene replacement”, “gene correction”, and “gene augmentation” [Katare and Aeri, 2010]. In replacement therapy, a normal gene is inserted somewhere in the genome so that its product could replace that of a defective gene. This approach may be suitable for recessive disorders, which are marked by deficiency of an enzyme or other proteins. Although, the gene functions in the genome providing an appropriate regulatory sequence, the approach may not be successful in treating dominant disorders associated with the production of an abnormal gene product, which interferes with the product of normal gene [Katare and Aeri, 2010]. Corrective gene therapy requires replacement of a mutant gene or a part of it with a normal sequence. This can be achieved by using recombinant technology. Another form of corrective therapy involves the suppression of a particular mutation by a transfer RNA that is introduced into a cell [Katare and Aeri, 2010]. In gene augmentation, introducing a normal genetic sequence into a host genome modifies the expression of mutant gene in defective cell and the defective host gene remains unaltered. In general, the gene therapy recipient cells may be germline cells or somatic cells. Germline cells therapy involves modifying the genes in germ cells which will pass these genetic changes to the future generations. Somatic cells therapy involves the insertion of genes into specific somatic cells like the bone marrow stem cells, fibroblasts, hepatocytes or mycocytes [Katare and Aeri, 2010]. This form of gene therapy is being used at most genetic engineering laboratories throughout the world. Clearly, gene therapy provides great opportunities for treating diseases from genetic disorders, infections and cancer [Park et al., 2006]. While the genetic mutations underlying various diseases are well understood, delivering a corrective gene to the unhealthy organs/tissues remains a remarkable challenge [Stone, 2010]. To achieve successful gene therapy, development of proper gene delivery systems could be one of the most important factors. Gene delivery systems should be designed to protect the genetic materials from premature degradation in systemic blood stream and to efficiently transfer the therapeutic genes to target cells. Intracellular delivery systems will be required for all molecules that have intracellular function. For example, nucleic acid molecules including encoding genes, oligonucleotides and RNA molecules must enter cells and target the nucleus when transcription is the target. Regardless of the molecules for delivery, a common requirement
منابع مشابه
Barriers and recent advances in non-viral vectors targeting the lungs for cystic fibrosis gene therapy
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in CFTR genes that affect chloride ion channel. The CF is a good nominee for gene therapy as the asymptomatic carriers are phenotypically normal, and the desired cells are accessible for vector delivery. Gene therapy shows promising effects involving the correction of gene or replacement of the mutant gene with the func...
متن کاملEvaluation of Cell Penetrating Peptide Delivery System on HPV16E7 Expression in Three Types of Cell Line
Background: The poor permeability of the plasma and nuclear membranes to DNA plasmids are two major barriers for the development of these therapeutic molecules. Therefore, success in gene therapy approaches depends on the development of efficient and safe non-viral delivery systems. Objectives: The aim of this study was to investigate the in vitro delivery of plasmid DNA encoding HPV16 E7 gene...
متن کاملنگاهی به ژن درمانی، پیشرفتهای اخیر و چشم انداز آینده
Human gene therapy has attracted increasing attention as a highly encouraging therapeutic approach to treat wide variety of diseases, other than genetically inherited and monogenic disorders. This approach entails the introduction and expression of a variety of nucleic acids into human target cells for therapeutic purposes. In this article, we review the history, highlights, recently progresses...
متن کاملDNA-Based Vaccine Is More Efficient than Non-Pathogenic Live Vaccine for the Prevention of HPV16 E7-Overexpressing Cancers
Introduction: Vaccinology provides promising approaches for the control of various infectious diseases. Among different strategies, DNA vaccines offer attractive research opportunities for development of vaccines for induction of antigen-specific immunity owing to their stability, simplicity of delivery, safety and cost effectiveness. However, there is a need to increase their potency by the ...
متن کاملNano-niosomes in drug, vaccine and gene delivery: a rapid overview
Niosomes, non-ionic surfactant vesicles (NSVs), are the hydrated lipids composed mainly of different classes of non-ionic surfactants, introduced in the seventies as a cosmetic vehicle. Nowadays, niosomes are used as important new drug delivery systems by many research groups and also they are effective immunoadjuvants which some commercial forms are available in the market. These vesicles rece...
متن کاملNon-biological gene carriers designed for overcoming the major extra- and intracellular hurdles in gene delivery, an updated review
Gene therapy as a modern therapeutic approach has not yet advanced to a globally-approved therapeutic approach. Lack of adequate reliable gene delivery system seems to be one of the major reasons from the pharmaceutical biotechnology point of view. Main obstacles delaying successful application of human gene therapy are presented in this review. The unique advantages of non-biological gene carr...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
عنوان ژورنال:
دوره شماره
صفحات -
تاریخ انتشار 2012