Genes, Vol. 10, Pages 289: In Vitro Gene Delivery in Retinal Pigment Epithelium Cells by Plasmid DNA-Wrapped Gold Nanoparticles

Genes, Vol. 10, Pages 289: In Vitro Gene Delivery in Retinal Pigment Epithelium Cells by Plasmid DNA-Wrapped Gold Nanoparticles Genes doi: 10.3390/genes10040289 Authors: Trigueros Domènech Toulis Marfany Many rare diseases course with affectation of neurosensory organs. Among them, the neuroepithelial retina is very vulnerable due to constant light/oxidative stress, but it is also the most accessible and amenable to gene manipulation. Currently, gene addition therapies targeting retinal tissue (either photoreceptors or the retinal pigment epithelium), as a therapy for inherited retinal dystrophies, use adeno-associated virus (AAV)-based approaches. However, efficiency and safety of therapeutic strategies are relevant issues that are not always resolved in virus-based gene delivery and alternative methodologies should be explored. Based on our experience, we are currently assessing the novel physical properties at the nanoscale of inorganic gold nanoparticles for delivering genes to the retinal pigment epithelium (RPE) as a safe and efficient alternative approach. In this work, we present our preliminary results using DNA-wrapped gold nanoparticles (DNA-gold NPs) for successful in vitro gene delivery on human retinal pigment epithelium cell cultures, as a proof-of-principle to assess its feasibility for retina in vivo gene delivery. Our results show faster expression of a reporter gene in cells transfected with DNA-gold NPs compared to DNA-liposome com...
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Article Source Type: research

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Source: Videocast - All Events - Category: General Medicine Tags: Upcoming Events Source Type: video
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA) are co-hosting a Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies. Current adeno-associated virus (AAV) manufacturing capacity cannot keep pace with the demand for AAV gene therapy clinical trials. The purpose of the meeting is to bring together thought leaders, key stakeholders and innovators to explore obstacles and identify opportunities for expanding the capacity of AAV manufacturing f...
Source: Videocast - All Events - Category: General Medicine Tags: Upcoming Events Source Type: video
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