Polymer‐mediated gene therapy: Recent advances and merging of delivery techniques

Abstract

The ability to safely and precisely deliver genetic materials to target sites in complex biological environments is vital to the success of gene therapy. Numerous viral and nonviral vectors have been developed and evaluated for their safety and efficacy. This study will feature progress in synthetic polymers as nonviral vectors, which benefit from their chemical versatility, biocompatibility, and ability to carry both therapeutic cargo and targeting moieties. The combination of synthetic gene carrying constructs with advanced delivery techniques promises new therapeutic options for treating and curing genetic disorders. This article is characterized under: Therapeutic Approaches and Drug Discovery > Nanomedicine for Oncologic Disease Therapeutic Approaches and Drug Discovery > Emerging Technologies

Advances in the Development and the Applications of Nonviral, Episomal Vectors for Gene Therapy

“…Polymer and liposome chemists increasingly collaborate with biologists to optimize the capability of synthetic nanostructures for improving nonviral gene delivery. Significant advances emerging from these partnerships include novel polymer designs for polyplex formation, targeted systems with enhanced efficacy, and advanced synthetic constructs with improved stability when interacting with proteins and erythrocytes in blood 14 . More recently, novel PEGylated siRNA-loaded LNPs have addressed the greatest challenge in implementing siRNA therapeutics, which is their delivery 15 .…”

Therapeutic Applications of Genes and Gene-Engineered Mesenchymal Stem Cells for Femoral Head Necrosis

“…100,101 To enhance the safety of this approach, significant effort is focused on the improvement of virus-packaging systems, development of the suicide gene strategy, and pre-detection of insertion mutations before transplantation. 98,[100][101][102] Incorporation of a suicide gene to viral vector, which allows destroying the residual delivering cells after the therapeutic factor has been delivered, is highly attractive in cell-based gene therapy.…”

“…Recent advances in material sciences have led to the development of a series of new synthetic polymer-and lipid-based non-viral delivery systems with improved gene transfer efficiency, such as polymers, cationic lipids, and virus-like particles. 102,103 Nevertheless, the non-integration vectors, including non-viral vectors, adenovirus, AAV, baculovirus, and others, provide only transient gene expression. It is also necessary to explore safer and more efficient techniques of genetic manipulation.…”

Peptides as key components in the design ofnon‐viralvectors for gene delivery

“…Polymers comprised of positively charged moieties that are capable of condensing DNA into complexes known as polyplexes, have garnered significant attention in the field of gene therapy. [89,90] Decoration of bioactive peptides on these polymeric scaffolds generates physiologically stable complexes and enhances gene transfer efficacy by imparting multifunctional properties to the polyplexes. This can be done through bioactive peptides bearing fusogenic, stimuli responsive, or targeting moieties.…”