Non-Viral Carriers for Nucleic Acids Delivery: Fundamentals and Current Applications

SIMPLE SUMMARY: Gene therapy is one of the most promising approaches to treating various inherited and acquired diseases by delivering genetic constructs into the cells and tissues of the human body. Genetic constructs in general can be described as different DNA and RNA molecules thatcan encode therapeutic proteins or may influence their expression. However, cells and tissues cannot be penetrated easily by DNA and RNA because of their high molecular weight and negative charge. For this purpose, various gene delivery vehicles are studied. The most efficient vehicles are viruses, but they can be dangerous for the organism, so alternate non-viral approaches are intensively studied. This review summarizes recent information on non-viral carriers and the basic requirements for their development. ABSTRACT: Over the past decades, non-viral DNA and RNA delivery systems have been intensively studied as an alternative to viral vectors. Despite the most significant advantage over viruses, such as the lack of immunogenicity and cytotoxicity, the widespread use of non-viral carriers in clinical practice is still limited due to the insufficient efficacy associated with the difficulties of overcoming extracellular and intracellular barriers. Overcoming barriers by non-viral carriers is facilitated by their chemical structure, surface charge, as well as developed modifications. Currently, there are many different forms of non-viral carriers for various applications. This review aimed to summarize recent developments based on the essential requirements for non-viral carriers for gene therapy.