Applications of lipid nanoparticles in gene therapy.
Solid lipid nanoparticles (SLNs) and nanostructured lipid carriers (NLCs) have been recognized, among the large number of non-viral vectors for gene transfection, as an effective and safety alternative to potentially treat both genetic and not genetic diseases. A key feature is the possibility to be designed to overcome the numerous challenges for successful gene delivery. Lipid nanoparticles (LNs) are able to overcome the main biological barriers for cell transfection, including degradation by nucleases, cell internalization intracellular trafficking, and selectively targeting to a specific cell type. Additionally, they present important advantages: from a safety point of view LNs are prepared with well tolerated components, and from a technological point of view, they can be easily produced at large-scale, can be subjected to sterilization and lyophilization, and have shown good storage stability. This review focuses on the potential of SLNs and NLCs for gene therapy, including the main advances in their application for the treatment of ocular diseases, infectious diseases, lysosomal storage disorders and cancer, and current research for their future clinical application.
['Del Pozo-Rodríguez|Ana|A|', 'Solinís|María Ángeles|MÁ|', 'Rodríguez-Gascón|Alicia|A|']
D000818:Animals D002462:Cell Membrane D002467:Cell Nucleus D003141:Communicable Diseases D004337:Drug Carriers D011992:Endosomes D005128:Eye Diseases D000795:Fabry Disease D005260:Female D005612:Freeze Drying D015316:Genetic Therapy D006801:Humans D007107:Immune System D008055:Lipids D016464:Lysosomal Storage Diseases D008297:Male D051379:Mice D050997:Nanomedicine D053758:Nanoparticles D009369:Neoplasms D010316:Particle Size D041441:Retinoschisis
Gene therapy Solid lipid nanoparticles Nanostructured lipid carriers Non-viral vectors Infectious diseases Cancer Fabry disease X-linked juvenile retinoschisis