Functionalized lipoplexes and polyplexes for cancer therapy

Abstract

Gene therapy has gained acclaim in recent years due to its ability to treat a variety of diseases, particularly those caused by oncological and genetic abnormalities such as cancer, which is characterized by mutations in cancer-critical genes. Gene therapy provides the ability to supplement, repair, or silence such defective genes by providing therapeutic nucleic acids (NAs) to patients, as well as stimulating their immune systems to combat the disease. Despite the challenges involved in gene therapy for cancer, the latter approach (a form of immunotherapy) is showing promising results in early-stage clinical trials. The success of gene therapy is entirely dependent on the development of nontoxic and highly effective gene transport methods. In recent years, new lipid and polymer-based transport systems have emerged to shield oligonucleotides from damage, facilitate their entry to target cells, and promote an efficient endosomal release. The most promising approaches for the complexation of NAs with lipids (Lipoplexes) and different kinds of polymers (Polyplexes) are discussed in this chapter. The advantages and disadvantages of each method, as well as the possibility of using lipids and polymers in a single formulation, will also be discussed, followed by the future prospects for gene therapy involving nanotechnology—an approach that seems to be somewhat promissory—bringing significant new insights into the treatment of many diseases. © 2024 Elsevier Inc. All rights reserved.