<i>In vivo</i> Gene Therapy to the Liver and Nervous System: Promises and Challenges.

Front Med (Lausanne) · 2021 · PMC8803894 · PMID 35118085

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a decreasing trendno p-value reported

In vivo GT to the Liver Systemic administration of AAV vectors expressing coagulation factor VIII or IX transgene in hepatocytes is in advanced phase of clinical testing as a treatment for the inherited coagulation disorder hemophilia and showed multi-year reconstitution of therapeutic amounts of the clotting factors, even though a decreasing trend in factor VIII activity has been reported ( 18 – 20 ).

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