Publications by authors named "M E McClements"
Uveitis is characterised by breakdown of the blood-retinal barrier (BRB), allowing infiltration of immune cells that mediate intraocular inflammation, which can lead to irreversible damage of the neuroretina and the loss of sight. Treatment of uveitis relies heavily on corticosteroids and systemic immunosuppression due to limited understanding of disease pathogenesis. We performed single-cell RNA-sequencing of retinas, as well as bulk RNA-sequencing of retinal pigment epithelial (RPE) cells from mice with experimental autoimmune uveitis (EAU) versus healthy control.
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- Conventional gene therapy has limitations like only treating loss-of-function diseases and being restricted by viral packaging sizes, which prevents addressing larger genes.
- The advent of CRISPR/Cas technology marks a significant shift in genetic therapy, allowing for precise gene editing and a broader range of treatable diseases, including advancements like base and prime editing.
- The review covers the mechanisms of CRISPR-activators (CRISPRa), their current applications in vivo, and explores challenges in translating this technology, particularly in the context of eye diseases.
Article Synopsis
- * Though some therapies have had successful outcomes, others have failed to meet key goals, leading to learnings that will improve future clinical trial designs.
- * Ongoing research and trials aim to refine these treatments, which could dramatically change how retinal disorders are managed, potentially restoring vision and enhancing quality of life for many patients.
Age-related macular degeneration (AMD) is the most common cause of untreatable blindness in the developed world. Recently, CDHR1 has been identified as the cause of a subset of AMD that has the appearance of the "dry" form, or geographic atrophy. Biallelic variants in CDHR1-a specialized protocadherin highly expressed in cone and rod photoreceptors-result in blindness from shortened photoreceptor outer segments and progressive photoreceptor cell death.
View Article and Find Full Text PDFAge-related macular degeneration (AMD) is the leading cause of irreversible vision loss among the elderly in the developed world. Whilst AMD is a multifactorial disease, the involvement of the complement system in its pathology is well documented, with single-nucleotide polymorphisms (SNPs) in different complement genes representing an increased risk factor. With several complement inhibitors explored in clinical trials showing limited success, patients with AMD are still without a reliable treatment option.
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