Polymeric Nanoparticles for Gene Therapy

Sushma Pullela^*
*Correspondence: Sushma Pullela, Department of Biotechnology, Osmania University, Hyderabad, Telangana, India, Email:

Perspective

Polymeric nanoparticles (NPs) are small particles with a diameter of 1 to 1000 nm that can be loaded with active chemicals or surface-adsorbed onto the polymeric core. The term "nanoparticle" is used to describe both nanocapsules and nanospheres, which differ in their morphological structure. Polymeric NPs have showed considerable promise in the delivery of medications to specific locations for the treatment of a variety of ailments. The most generally utilised methods for the manufacture and characterisation of polymeric NPs, as well as the association efficiency of the active chemical to the polymeric core and in vitro release mechanisms, are discussed in this study. We also examine the toxicity and ecotoxicology of nanoparticles to humans and the environment because nanoparticle safety is a top topic.

Polymeric nanoparticles are rapidly advancing in a wide range of fields, including electronics, photonics, conducting materials, and sensors, as well as medicine, pollution management, and environmental technologies. Gene therapy has evolved as one of the most advanced applications of polymers in medicine, with the capacity to treat illnesses from the current period. However, there are various hurdles to gene transfer in biological systems that polymer engineering can help overcome. The effectiveness of the delivery vehicle or vector is one of the most critical difficulties. Because of their low toxicity, potential for targeted administration, long-term stability, absence of immunogenicity, and low production cost, non-viral delivery methods have gotten a lot of attention in recent decades. Because of their ease of synthesis and flexibility, cationic polymers have emerged as attractive options for nonviral gene delivery systems. These polymers can be conjugated with genetic material via electrostatic attraction at physiological pH, thereby facilitating gene delivery.

The structure, molecular weight, and surface charge of cationic polymers all have an impact on their gene transfection efficacy. Synthetic polymers such as poly (l-lysine), poly (l-ornithine), linear and branching polyethyleneimine, diethylaminoethyl-dextran, poly (amidoamine) dendrimers, and poly (amidoamine) dendrimers are notable examples of polymers that have appeared in the previous decade for use in gene therapy (dimethylaminoethyl methacrylate). Natural polymers like chitosan, dextran, gelatin, and pullulan were studied, as well as synthetic analogues with advanced properties like guanidinylated bio-reducible polymers. This overview covers the history of polymers in medicine, as well as polymer synthesis methods, including top-down and bottom-up approaches. The importance of evaluating functionalization techniques for therapeutic and formulation stability is also emphasised. This study stands out as a one-stop synopsis of developments in the field, with an overview of the properties, difficulties, and functionalization options, as well as uses of polymeric delivery systems in gene therapy.

Polymeric nanoparticles characterization

Physical characteristics such as composition and concentration, as well as size, shape, surface properties, crystallinity, and dispersion state, can all affect polymeric NPs. These features are frequently evaluated using a variety of methodologies in order to fully characterise the NPs. Electron microscopy, dynamic light scattering (DLS) or photon correlation spectroscopy (PCS), near-infrared spectroscopy, electrophoresis, and chromatography are just a few of the most widely utilised techniques. Characterization of polymeric NPs is important not only for identifying their applicability, but also for determining issues like Nano toxicology and workplace exposure evaluation, both of which are required for assessing health and safety risks and controlling manufacturing processes. Polymeric nanoparticles (PNP) are rapidly growing in popularity and are used in a wide range of applications, including electronics, photonics, conducting materials, and sensors, as well as pollution control, environmental technology, and medicine. Gene therapy is a relatively new branch of medicine that has the potential to alleviate and cure many diseases that are resistant to regular treatment, and PNPs could play a key part in its advancement. Alnylam's siRNA medication Onpattro was recently licenced by the US Food and Drug Administration (FDA) for hereditary amyloidosis.

Onpattro incorporates the therapeutic siRNA moiety in a lipid nanoparticle (NP), which is delivered to the liver by infusion and prevents the body from manufacturing disease-causing proteins. However, much work needs to be done in order to overcome the hurdles to gene delivery. Virus vectors, while effective, have safety concerns, despite virologists' best attempts to reduce immunogenicity and side effects. Because of their low toxicity, potential for targeted administration, long-term stability, absence of immunogenicity, and low production cost, non-viral delivery systems have become popular.