New research led by the University of Bristol has raised concerns about the potential risks of gene therapy for treating incurable eye diseases, particularly for older women. Published in Molecular Therapy, the study highlights how both age and gender can influence the inflammatory responses triggered by these cutting-edge treatments, with implications for patient safety and therapy effectiveness.
Gene therapies have recently emerged as a promising solution for previously untreatable eye diseases. In the UK alone, approximately 2 million people suffer from sight loss, with more than a million affected by conditions like age-related macular degeneration, glaucoma, and diabetic eye disease—many of which currently have no cure. While gene therapies offer hope for restoring vision, clinical trials have reported an alarming side effect: unexpected inflammation that can lead to permanent vision loss in some patients.
The study, which focuses on the underlying causes of inflammation in gene therapy, aims to improve the safety and effectiveness of these treatments. Researchers have long been aware that inflammation can limit the success of gene therapies, but the new findings suggest that individual factors such as age and gender could play a crucial role in how the body responds to these treatments.
Gene therapy typically involves using a harmless virus, such as the Adeno-Associated Virus (AAV), to deliver therapeutic genes directly to eye cells, helping them regain their function and prevent further damage. The AAV virus is often recognized by the immune system as potentially harmful, which can trigger an inflammatory response. This immune reaction not only undermines the therapy’s effectiveness but may also prevent necessary adjustments to dosages, further complicating treatment outcomes.
To better understand how inflammation arises and how it can be managed, the Bristol team conducted experiments comparing immune responses in male and female animal models of varying ages. The results revealed significant differences in how the immune cells of older females reacted to the gene therapy. While younger male and female models exhibited a similar immune response, inflammation in older models was more pronounced and prolonged. In particular, older females showed a stronger inflammatory response linked to retinal damage, raising concerns about the long-term safety of gene therapies for this group.
Dr. Alison Clare, lead author of the study and Senior Research Associate at Bristol Medical School, emphasized the importance of these findings for future clinical practices. “Our research is the first to demonstrate that both age and sex significantly affect the risk of adverse inflammatory reactions to gene therapies in the eye. These insights underline the need for tailored treatment approaches that consider individual risk factors, ensuring better outcomes and minimizing potential harm,” she said.
The research highlights an urgent need for personalized gene therapy strategies that take into account the patient’s gender, age, and unique immune responses. For older women, who appear to be at a heightened risk of severe inflammatory reactions, these findings suggest that extra caution will be necessary to prevent damage and vision loss.
As gene therapy continues to evolve as a potential treatment for various eye disorders, the study calls for a more nuanced approach to patient selection and treatment protocols. By addressing the specific risks posed by age and gender, researchers hope to optimize these therapies and ensure that they are both safe and effective for all patients, especially vulnerable groups.
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