Condition: Hemophilia B, Severe · Hemophilia A, Severe · Sponsor: University College, London
To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer following AAV mediated gene transfer. This is to obtain tissue for analysis, to understand if FIX/FVIII transgenic protein expression is mediated by AAV proviral DNA that is integrated into the host cell DNA or if stable expression in humans is mediated by episomal maintained AAV genome.
This description comes directly from the study's public registry record.
Paul Batty · 020 7794 0500 · paul.batty@ucl.ac.uk
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| Royal Free Hospital | London, United Kingdom | Recruiting |
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Source record: clinicaltrials.gov/study/NCT04817462