Publications by authors named "Alexandria Forbes"
Purpose: To assess the safety and efficacy of AAV5-hRKp.RPGR in participants with retinitis pigmentosa GTPase regulator (RPGR)-associated X-linked retinitis pigmentosa (XLRP).
Design: Open-label, phase 1/2 dose escalation/expansion study (ClinicalTrials.
Variants within the Retinitis Pigmentosa GTPase regulator () gene are the predominant cause of X-Linked Retinitis Pigmentosa (XLRP), a common and severe form of inherited retinal disease. XLRP is characterised by the progressive degeneration and loss of photoreceptors, leading to visual loss and, ultimately, bilateral blindness. Unfortunately, there are no effective approved treatments for RPGR-associated XLRP.
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- A clinical trial assessed the safety and effectiveness of AAV8-hCARp.hCNGB3 gene therapy in treating CNGB3-associated achromatopsia (ACHM) in 23 participants, including adults and children.
- Participants received varying doses of the treatment in their worst-seeing eye, with corticosteroids given to manage any inflammation.
- While the therapy showed an acceptable safety profile, with mild to moderate inflammation in some cases, there were individual improvements in color vision and quality of life, suggesting potential benefits warranting further research.
Gap junctions coordinate processes ranging from muscle contraction to ovarian follicle development. Here we show that the gap junction protein Zero population growth (Zpg) is required for germ cell differentiation in the Drosophila ovary. In the absence of Zpg the stem cell daughter destined to differentiate dies.
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