Polymers and Nanogels for Gene Therapy

Dear Colleagues,

Gene therapy has attracted significant attention over the past few decades for the treatment of inherited and acquired diseases. Even with substantial effort in recent years, delivery of genes to the target cells remains a significant challenge and the successful clinical translation of these technologies are still very slim. Current research efforts are focused on designing and synthesizing proper vectors that can efficiently compact and protect oligonucleotides. However, due to the hydrophilic and negatively charged nature of DNA or RNA and their low stability in the physiological environment (free oligonucleotides and plasmid DNA are rapidly degraded by serum nucleases in the blood when injected intravenously), the development of suitable carriers has been a daunting task for researchers working in this area. In addition, since both the genes and cell surfaces are negatively charged; entry of free DNA or RNA inside cells is a difficult task. So, the clinical translation of gene therapy is heavily dependent on the development of safe and efficient gene delivery systems. Research initially focused on viral vectors as these vectors exhibited high efficiency at delivering both DNA and RNA to numerous cell lines. However, several problems such as toxicity, immunogenicity and limitations with respect to scale-up procedures associated with viral vector systems have encouraged the investigation of synthetic DNA or RNA carriers into targeted tissue. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are the preferred means for compacting DNA for systemic delivery, but, unlike viral analogues, non-viral gene carriers reliably exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles, and the challenges are significant in vivo. However, their tunable biocompatibility and facilitated production make them potentially useful and attractive for gene therapy. As a result, major research efforts have focused on designing cationic systems that can form complexes with DNA, and avoid both in vitro and in vivo barriers.

This Special Issue entitled “Polymers and Nanogels for Gene Therapy” will cover recent developments in the use of precisely engineered polymers, nanoparticles and nanogels that can efficiently condense DNA and RNA and that demonstrate excellent gene expression or gene knock down efficacies. Special efforts in the design of new materials that are safe and efficient will be highlighted. Manuscripts on modification of polymers and nanoparticles to improve the gene expression or gene knock down efficacies as well as topics on mechanistic studies are also welcome. Contributions from both original research studies and comprehensive reviews will be considered.

Prof. Dr. Ravin Narain
Guest Editor

Manuscript Submission Information

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Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-anonymized peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Polymers is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2700 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.