Browsing by Subject "gene therapy"
Now showing items 1-5 of 5
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Controlled Non‐Viral Gene Delivery in Cartilage and Bone Repair: Current Strategies and Future Directions
(2018)Recent advances in the approval and commercialization of gene therapeutics have fostered the return of gene therapy to center stage. But despite new optimism, no Food and Drug Administration approved product exists for the ... -
Development of gene therapies for retinal degenerations
(Trinity College Dublin. School of Genetics & Microbiology. Discipline of Genetics, 2021)The studies described in this thesis explore potential gene therapies for retinal degenerative diseases using adeno-associated virus (AAV) vectors for gene delivery. Inherited retinal degenerations (IRDs) are a leading ... -
Exploration of gene therapies targeting oxidative stress and NAD+ pathways for the treatment of optic neuropathy
(Trinity College Dublin. School of Genetics & Microbiology. Discipline of Genetics, 2022)The World Health Organisation estimates that there are approximately 300 million people suffering from some form of blindness, from inherited retinal degenerations to complex diseases such as glaucoma. The vast heterogeneity ... -
Intravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy.
(2013)Leber hereditary optic neuropathy (LHON) is a mitochondrially inherited form of visual dysfunction caused by mutations in several genes encoding subunits of the mitochondrial respiratory NADH-ubiquinone oxidoreductase ... -
RPE-Directed Gene Therapy Improves Mitochondrial Function in Murine Dry AMD Models
(2023)Age-related macular degeneration (AMD) is the most common cause of blindness in the aged population. However, to date there is no effective treatment for the dry form of the disease, representing 85–90% of cases. AMD is ...