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    AAV8 gene therapy for mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) in a patient's liver maintained ex situ for 9 days

    AAV8 gene therapy for mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) in a patient's liver maintained ex situ for 9 days by Teresa Brevini, Lisa Swift, Helen Reynolds, John Ong, Richard Stopforth, Yogeshkumar Malam, Harry Spiers, AnnaPaterson, Adam Duckworth, Arthur Kaser, Vassillis Kosmoliaptsis, Ramon Marti, Jelle van den Ameele, Gwilym Webb, Christopher Watson, Andrew Butler, Fotios Sampaziotis

    Published 2025-07-01
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