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Description
For instance, in a gene therapy trial for haemophilia B, an AAV serotype 2 vector containing the human Factor IX transgene delivered via hepatic artery infusion, led to a transient therapeutic level of transgene expression lasting 8 weeks due to destruction of transduced hepatocytes by an AAV capsid-specific CD8 + T cell response [109]
Study limitations and conclusion Several factors contributed to our failure to reach the targeted participant number, including in-house deliveries restricted to working hours

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