Browsing by Subject "AAV"
Now showing items 1-8 of 8
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AAV-Delivered Tulp1 Supplementation Therapy Targeting Photoreceptors Provides Minimal Benefit in Tulp1−/− Retinas
(2020)With marketing approval of the first ocular gene therapy, and other gene therapies in clinical trial, treatments for inherited retinal degenerations (IRDs) have become a reality. Biallelic mutations in the tubby like protein ... -
Claudin-5 expression at the Blood Brain Barrier and its therapeutic potential in neurological disease
(Trinity College Dublin. School of Genetics & Microbiology. Discipline of Genetics, 2023)Breakdown of the blood brain barrier's (BBB) integrity is a hallmark pathology in numerous neurological disorders. However, while research and therapeutic development has mostly focussed on neuronal function, the high ... -
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 ... -
Novel therapeutic approaches for open-angle glaucoma
(Trinity College Dublin. School of Genetics & Microbiology. Discipline of Genetics, 2021)Glaucoma is the leading cause of blindness worldwide after cataracts and it is estimated that the global prevalence of glaucoma will increase to 111.8 million by 2040 (1, 2). Primary open-angle glaucoma (POAG) is characterized ... -
Optimisation of AAV-NDI1 Significantly Enhances Its Therapeutic Value for Correcting Retinal Mitochondrial Dysfunction
(2023)AAV gene therapy for ocular disease has become a reality with the market authorisation of LuxturnaTM for RPE65-linked inherited retinal degenerations and many AAV gene therapies currently undergoing phase III clinical ... -
Optimized OPA1 Isoforms 1 and 7 Provide Therapeutic Benefit in Models of Mitochondrial Dysfunction
(2020)Optic Atrophy 1 (OPA1) is a mitochondrially targeted GTPase that plays a pivotal role in mitochondrial health, with mutations causing severe mitochondrial dysfunction and typically associated with Dominant Optic Atrophy ... -
Primary open-angle glaucoma: on the development of novel therapeutic approaches
(Trinity College Dublin. School of Genetics & Microbiology. Discipline of Genetics, 2018)Glaucoma is one of the most prevalent forms of preventable blindness, affecting more than 60 million people worldwide. While normotensive forms of the disease do exist, the majority of cases are caused by elevations in ... -
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 ...