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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Current status in paediatric liver transplantation for inherited metabolic diseases
Nedim Hadzic1, Carlo Dionisi-Vici2, Roshni Vara1,3
1Paediatric Liver, GI and Nutrition Service, King's College Hospital, London, UK.
Abstract:
Liver transplantation (LT) has evolved over the past few decades and is now an established therapy for several inherited metabolic diseases (IMDs) with overall good long-term outcomes. IMDs are now the second most common indication for paediatric LT with increasing waiting times and expanding indications. There have been innovations in surgical techniques and graft viability over the years to increase the donor pool. This group of patients poses several challenges including the increasing complexity of diseases considered amenable to LT, survival into adulthood, metabolic and neurocognitive monitoring post-LT. Following an international meeting of experts, we present a summary of current views and trends in paediatric LT for IMDs. We highlight that LT continues to offer a realistic alternative therapeutic approach despite the potential advancement in novel therapies.
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