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.
In a groundbreaking study published in Nature Communications [1], researchers at the Fred Hutchinson Cancer Center have reported encouraging findings from pre-clinical studies on an experimental gene therapy for genital and oral herpes infections caused by the herpes simplex virus (HSV).
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