Gene therapy for inborn errors of liver metabolism: progress towards clinical applications.
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ABSTRACT:
SUBMITTER: Brunetti-Pierri N
PROVIDER: S-EPMC2603013 | biostudies-literature | 2008
REPOSITORIES: biostudies-literature
Publications
Gene therapy for inborn errors of liver metabolism: progress towards clinical applications.
Italian journal of pediatrics 20081118 1
The treatment for inborn errors of liver metabolism is based on dietary, drug, and cell therapies (orthotopic liver transplantation). However, significant morbidity and mortality still remain, and alternative strategies are needed. Gene replacement therapy has the potential of providing a definitive cure for patients with these diseases. Significant progress has been made in the pre-clinical arena and achievement of efficacy in different animal models has been reported using multiple gene transf ...[more]
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