Non-viral nucleic acid delivery methods

Abstract

Delivery of nucleic acid-based molecules in human cells is a highly studied approach for the treatment of several disorders including monogenic diseases and cancers. Non-viral vectors for DNA and RNA transfer, although in general less efficient than virus-based systems, are particularly well adapted mostly due to the absence of biosafety concerns. Non-viral methods could be classified in two main groups: physical and vector-assisted delivery systems. Both groups comprise several different methods, none of them universally applicable. The choice of the optimal method depends on the predefined objectives and the features of targeted micro-environment. Areas covered: In this review, the authors discuss non-viral techniques and present recent therapeutic achievements in ex vivo and in vivo nucleic acid delivery by most commonly used techniques while emphasizing the role of 'biological particles', namely peptide transduction domains, virus like particles, gesicles and exosomes. Expert opinion: The number of available non-viral transfection techniques used for human therapy increased rapidly, followed by still moderate success in efficacy. The prospects are to be found in design of multifunctional hybrid systems that reflect the viral efficiency. In this respect, biological particles are very promising.

Cytokines in Pain: Harnessing Endogenous Anti-Inflammatory Signaling for Improved Pain Management

“…Specifically, viral approaches rely on viral vectors to mediate cellular transfection and subsequent gene expression. In contrast, non-viral gene therapies gain cellular access through a wide variety of mechanisms ranging from simple cellular uptake of naked plasmid DNA, to more formal packaging of genetic material within carriers like liposomes, polymers, or basic proteins to facilitate transfection (69)(70)(71)(72). These packaging modifications are often aimed at limiting immune detection, protecting the payload (e.g., plasmid DNA) from degradation by endonucleases, targeting to the therapy to cells of interest, as well as promoting cellular entry (73).…”

“…While non-viral IL-10 gene therapy has been shown to effectively relieve pathological pain in numerous animal models, initial studies required repeated large doses of DNA, thereby making clinical translation less feasible. To ameliorate the dose limitations, various carriers for non-viral gene therapy have been explored but are limited by adverse effects or added levels of complexity (71,72,141). Co-delivered adjuvants may offer a simpler alternative to carrier-type approaches.…”

Efficient DNA Condensation by a C3‐Symmetric Codeine Scaffold

“…Synthetic non‐viral vectors offer a number of advantages over viral systems such as improved safety, lack of immunogenicity, and ease of large‐scale production . Since they are amenable to engineering, these systems allow for flexibility with regard to the size of the gene being administered and can offer improved targeting effects with the ultimate aim of delivering more copies of genomic material to the requisite cell …”

“…[8] Since they are amenable to engineering, these systems allow for flexibility with regard to the size of the gene being administered and can offer improved targeting effects with the ultimate aim of delivering more copies of genomic material to the requisite cell. [8][9][10][11] This work focuses on the non-analgesic use of opioids as a bio-renewable resource capable of packaging DNA. While opioids are predominantly used for their potent analgesic properties, serving as one of the major drug classes within the pharmaceutical industry, [12,13] specific architectures were recently identified to have DNA binding properties.…”

Gene therapy as advanced technology for the treatment of Leukemia: CAR-T therapy

“…Gene gun and electroporation are the commonly used techniques. Lipoplexes and oligonucleotides are also used in treatment of some diseases (Slivac et al, 2017;Figure 1 . Kaji EHet al, 2001;) Cancer is a disease occurs due to abnormal growth of cells as well as their ability to spread from one body parts to the other body parts (Swisher et al, 2017).…”