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These findings indicate that the Ps-like disease in DKO* mice affects the adipose tissue and is associated with altered local and systemic lipid metabolism

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In utero gene therapy (IUGT) using GLOBE lentiviral vector phenotypically corrects the heterozygous humanised mouse model and its progress can be monitored using MRI techniques

Some patients will respond to 10% parenteral amino acid solutions (Aminosyn, Abbott Laboratories, Chicago) given at a dose of 500 mL over 8 to 12 hours intravenously through a large-bore central venous catheter