Novel gene delivery systems

Abstract

Gene therapy is an emerging field in medical and pharmaceutical sciences because of its potential in treating chronic diseases like cancer, viral infections, myocardial infarctions, and genetic disorders. Application of gene therapy is limited because of lack of suitable methods for proper introduction of genes into cells and therefore, this is an area of interest for most of the researchers. To achieve successful gene therapy, development of proper gene delivery systems could be one of the most important factors. Several nonviral and viral gene transfer methods have been developed. Even though the viral agents have a high transferring efficiency, they are difficult to handle due to their toxicity. To overcome the safety problems of the viral counterpart, several nonviral in vitro and in vivo gene delivery systems are developed. Out of these, the most promising and latest systems include polymer-based nonviral gene carriers, dendrimers, and physical means like electroporation, microinjection, etc., Shunning of possible immunogenicity and toxicity, and the feasibility of repeated administration are some of the merits of nonviral gene delivery systems over viral gene delivery. An ideal nonviral gene carrying system should possess all these merits without any compromise to its gene transferring efficiency. The viral gene delivery systems include lytic and nonlytic vectors for drug delivery. Inspite of its toxicity they are still preferred because of their long term expression, stability, and integrity. This review explores the recent developments and relevancy of the novel gene delivery systems in gene therapy.

Different strategies of gene delivery for treatment of cancer and other disorders

“…Major disadvantage is that it induces greater immune responses than microinjection. [18,20] Figure 1A shows the use of gene gun for gene therapy against cancer in clinical trials.…”

“…Indeed, the efficiency of this system depends on concentration and size of plasmid DNA, the intensity of the pulses, frequency and duration. [11,20,21] Photoporation: Photoporation method uses a single laser pulse using generation of transient pores to transfer plasmid DNA into the cells. The data showed that the level of transgene expression is similar to that of electroporation.…”

“…[18,19] Microinjection: In microinjection, cell membrane or nuclear membrane is penetrated by simple mechanical force using a needle of 0.5 µm-5 µm diameter. [5,20] This gene delivery system is mainly used to inject DNA constructs in vivo. [21] Jet injection: In jet injection method, mechanical compression is utilized to move fluid containing DNA without using any needle for injection of particles.…”

“…[18] Balistic DNA injection or gene gun: The particle bombardment or gene gun uses heavy metal microparticles (e.g., gold or tungsten, 1 µm-5 µm in diameter) to penetrate the target cells. [20][21][22] This method has two major advantages such as safety, and high efficiency against parenteral injection. The advantages of this approach are included: 1) total amount of DNA required for delivery is low, 2) no receptor is required, 3) size of DNA is not a problem, and 4) production of DNA-coated metal particles is easy to generate.…”

Approaches to Pharmaceutical Analysis of Modern Peptide and Oligonucleotide Products as Illustrated by a Small Interfering RNA-Based Novel Therapeutic for the Treatment of Bronchial Asthma

“…Поэтому главным препятствием для внедрения подобных препаратов в медицинскую практику является отсутствие эффективных и нетоксичных средств доставки генетических конструкций в клетки-мишени. Исследованиям по созданию систем доставки уделяется большое внимание [6].…”