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Vector challenge protocol. All subjects selected for postnatal VC were screened for anti-AAV binding (Ab) and NAbs underwent a prechallenge liver biopsy and were given 2 × 1011 vg/kg intravenously. All animals tolerated the additional dose of vector with no clinically overt immunologic responses. e5002 and e5003 died from anesthetic complications during the postchallenge liver biopsies.
