In an interview, Dr Zheng-Yi Chen reveals how his team’s single-dose CRISPR therapy successfully restored hearing and balance in adult mice with DFNA41 genetic deafness, marking a potential breakthrough for treating progressive hearing loss in humans.
New research from the Medical University of Vienna has identified a mechanism explaining why people with haemophilia A rarely experience oral bleeding, despite their blood clotting deficiency. The finding could have implications for understanding bleeding patterns in different coagulation disorders.
A groundbreaking RNA-based technology allows therapeutic genes that are too large for conventional delivery methods to be successfully transported into cells, offering new hope for treating muscular dystrophies and other genetic disorders caused by mutations in large genes.
A single-dose gene therapy has demonstrated significant reduction in bleeding episodes for adults with haemophilia B, potentially offering a long-term solution to this genetic disorder.
A groundbreaking gene therapy for Duchenne muscular dystrophy (DMD) has demonstrated significant potential in not only arresting muscle decline but also possibly repairing damaged muscle tissue. This novel treatment, developed by researchers at UW Medicine, utilises a series of protein packets delivered via viral vectors to replace the defective DMD gene within affected muscles.
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