The paper reports the case of two monozygous twins affected by LI-MLD undergoing HSC-GT with CD34+ cells transduced with a lentiviral vector encoding for the ARSA gene who were subsequently discovered to also harbour a MCP gene mutation; while the first transplanted twin developed severe VOD and TMA, DF prophylaxis and reduction of busulfan regimen were associated with absence of these complications in the second twin.
Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy
Morena, Francesco;
2018-01-01
Abstract
The paper reports the case of two monozygous twins affected by LI-MLD undergoing HSC-GT with CD34+ cells transduced with a lentiviral vector encoding for the ARSA gene who were subsequently discovered to also harbour a MCP gene mutation; while the first transplanted twin developed severe VOD and TMA, DF prophylaxis and reduction of busulfan regimen were associated with absence of these complications in the second twin.File in questo prodotto:
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