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- Gene_therapy_of_the_human_retina abstract "Retinal gene therapy holds great promise in treating different forms of non-inherited and inherited blindness.In 2008, three independent research groups reported that patients with the rare genetic retinal disease Leber's Congenital Amaurosis had been successfully treated using gene therapy with adeno-associated virus (AAV). In all three studies, an AAV vector was used to deliver a functional copy of the RPE65 gene, which restored vision in children suffering from LCA. These results were widely seen as a success in the gene therapy field, and have generated excitement and momentum for AAV-mediated applications in retinal disease.In retinal gene therapy, the most widely used vectors for ocular gene delivery are based on adeno-associated virus. The great advantage in using adeno-associated virus for the gene therapy is that it poses minimal immune responses and mediates long-term transgene expression in a variety of retinal cell types. For example tight junctions that form the blood-retina barrier, separate subretinal space from the blood supply, providing protection from microbes and decreasing most immune-mediated damages.".
- Gene_therapy_of_the_human_retina wikiPageID "27110987".
- Gene_therapy_of_the_human_retina wikiPageRevisionID "595627420".
- Gene_therapy_of_the_human_retina subject Category:Emerging_technologies.
- Gene_therapy_of_the_human_retina subject Category:Gene_delivery.
- Gene_therapy_of_the_human_retina subject Category:Medical_genetics.
- Gene_therapy_of_the_human_retina subject Category:Medical_research.
- Gene_therapy_of_the_human_retina subject Category:Molecular_biology.
- Gene_therapy_of_the_human_retina comment "Retinal gene therapy holds great promise in treating different forms of non-inherited and inherited blindness.In 2008, three independent research groups reported that patients with the rare genetic retinal disease Leber's Congenital Amaurosis had been successfully treated using gene therapy with adeno-associated virus (AAV). In all three studies, an AAV vector was used to deliver a functional copy of the RPE65 gene, which restored vision in children suffering from LCA.".
- Gene_therapy_of_the_human_retina label "Gene therapy of the human retina".
- Gene_therapy_of_the_human_retina sameAs m.0bs10xb.
- Gene_therapy_of_the_human_retina sameAs Q4682229.
- Gene_therapy_of_the_human_retina sameAs Q4682229.
- Gene_therapy_of_the_human_retina wasDerivedFrom Gene_therapy_of_the_human_retina?oldid=595627420.
- Gene_therapy_of_the_human_retina isPrimaryTopicOf Gene_therapy_of_the_human_retina.