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α-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice
(MDPI, 2021-05-21)
Fabry disease (FD) is a monogenic X-linked lysosomal storage disorder caused by a deficiency in the lysosomal enzyme α-Galactosidase A (α-Gal A). It is a good candidate to be treated with gene therapy, in which moderately ...
Nanopartículas lipídicas como sistemas de administración de sustancias activas: aspectos tecnológicos y aplicaciones terapéuticas
(2020)
Con los avances tecnológicos en los últimos años, las nanopartículas han demostrado poseer un enorme potencial para su aplicación clínica. Los sistemas lipídicos son los de uso más extendido debido a que presentan propiedades ...
Development of a stem cells-based gene therapy approach mediated by non-viral vectors with potential clinical applications in regenerative medicine
(2020-12-15)
En la tesis actual, demostramos que los nioplexos DP80 podían transfectar eficientemente célulasmesenquimales MSC D1 con los plásmidos pCMS-EGFP y pUNO1-hBMP7. Sin embargo, se requiere laoptimización del proceso de formulación ...
Topical Administration of SLN-Based Gene Therapy for the Treatment of Corneal Inflammation by De Novo IL-10 Production
(MDPI, 2020-06-23)
One of the main challenges in gene therapy is the issue of delivery, and it is especially relevant for the success of gene therapy in the cornea. In the present work, eye drops containing biocompatible non-viral vectors ...
Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives
(MDPI, 2020-02-20)
The use of messenger RNA (mRNA) in gene therapy is increasing in recent years, due to its unique features compared to plasmid DNA: Transient expression, no need to enter into the nucleus and no risk of insertional mutagenesis. ...
Niosome-Based Approach for In Situ Gene Delivery to Retina and Brain Cortex as Immune-Privileged Tissues
(MDPI, 2020-02-25)
Non-viral vectors have emerged as a promising alternative to viral gene delivery systems due to their safer profile. Among non-viral vectors, recently, niosomes have shown favorable properties for gene delivery, including ...
Correlation between Biophysical Properties of Niosomes Elaborated with Chloroquine and Different Tensioactives and Their Transfection Efficiency
(MDPI, 2021-10-26)
Lipid nanocarriers, such as niosomes, are considered attractive candidates for non-viral gene delivery due to their suitable biocompatibility and high versatility. In this work, we studied the influence of incorporating ...
How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy?
(MDPI, 2021-07-14)
Efficient delivery of genetic material into cells is a critical process to translate gene therapy into clinical practice. In this sense, the increased knowledge acquired during past years in the molecular biology and ...
Selection and characterization of novel malolactic starters from Rioja Alavesa region: in the pursuit of free-histamine wine
(2021-04-15)
Biogenic amines (BAs) are considered a high priority issue for wine industry in terms of product quality and safety. They may cause several adverse symptoms which are enhance in susceptible individuals. The inoculation of ...
MMP-9 Downregulation with Lipid Nanoparticles for Inhibiting Corneal Neovascularization by Gene Silencing
(MDPI, 2019-04-18)
Gene silencing targeting proangiogenic factors have been shown to be a useful strategy in the treatment of corneal neovascularization (CNV). Among interference RNA (RNAi) molecules, short-hairpin RNA (shRNA) is a plasmid-coded ...