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

Introduction: Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a rare genetic disorder caused by mutations in TYMP.1 This results in nucleoside accumulation, mitochondrial damage and progressive gastrointestinal and neurological dysfunction leading to cachexia and death before 40 yea...

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Main Authors: 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
Format: Article
Language:English
Published: Elsevier 2025-07-01
Series:Clinical Medicine
Online Access:http://www.sciencedirect.com/science/article/pii/S1470211825000739
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