New research shows epigenetic silencing may limit durability of AAV gene therapy in haemophilia by reducing long term transgene expression in liver cells.
Jessica Nance, MD, MS, discusses maximizing gene therapy efficiency by selecting vectors for gene size, tissue targeting, and low exposure, and focusing on precise promoters for expression. This is a ...
Gene and cell therapy is moving fast – and in a clear direction. Programs are pushing toward more complex payloads, virus-free engineering, and manufacturing models that can scale from early research ...
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