title: Applications of lipid nanoparticles in gene therapy.
abstract: 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.
authors: Pharmacokinetic, Nanotechnology and Gene Therapy Group (PharmaNanoGene), Faculty of Pharmacy, Centro de investigación Lascaray ikergunea, University of the Basque Country UPV/EHU, Paseo de la Universidad, 7, 01006 Vitoria-Gasteiz, Spain.
authors: Ana
authors: A
authors: Del Pozo-Rodríguez
authors: Pharmacokinetic, Nanotechnology and Gene Therapy Group (PharmaNanoGene), Faculty of Pharmacy, Centro de investigación Lascaray ikergunea, University of the Basque Country UPV/EHU, Paseo de la Universidad, 7, 01006 Vitoria-Gasteiz, Spain.
authors: María Ángeles
authors: MÁ
authors: Solinís
authors: Pharmacokinetic, Nanotechnology and Gene Therapy Group (PharmaNanoGene), Faculty of Pharmacy, Centro de investigación Lascaray ikergunea, University of the Basque Country UPV/EHU, Paseo de la Universidad, 7, 01006 Vitoria-Gasteiz, Spain. Electronic address: alicia.rodriguez@ehu.eus.
authors: Alicia
authors: A
authors: Rodríguez-Gascón
mesh_terms: D000818:Animals; D002462:Cell Membrane / Q000378:metabolism; D002467:Cell Nucleus / Q000378:metabolism; D003141:Communicable Diseases / Q000235:genetics / Q000628:therapy; D004337:Drug Carriers / Q000737:chemistry; D011992:Endosomes / Q000378:metabolism; D005128:Eye Diseases / Q000235:genetics / Q000628:therapy; D000795:Fabry Disease / Q000235:genetics / Q000628:therapy; D005260:Female; D005612:Freeze Drying; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D007107:Immune System; D008055:Lipids / Q000737:chemistry*; D016464:Lysosomal Storage Diseases / Q000235:genetics / Q000628:therapy; D008297:Male; D051379:Mice; D050997:Nanomedicine / Q000379:methods*; D053758:Nanoparticles / Q000737:chemistry*; D009369:Neoplasms / Q000628:therapy; D010316:Particle Size; D041441:Retinoschisis / Q000235:genetics / Q000628:therapy
chemical_list: D004337:Drug Carriers; D008055:Lipids
keywords: Cancer; Fabry disease; Gene therapy; Infectious diseases; Nanostructured lipid carriers; Non-viral vectors; Solid lipid nanoparticles; X-linked juvenile retinoschisis