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Brunetti-Pierri N. Gene therapy for inborn errors of liver metabolism: progress towards clinical applications. Ital J Pediatr. 2008;34(1):2doi: 10.1186/1824-7288-34-2.
Brunetti-Pierri, N. (2008). Gene therapy for inborn errors of liver metabolism: progress towards clinical applications. Italian journal of pediatrics, 34(1), 2. https://doi.org/10.1186/1824-7288-34-2
Brunetti-Pierri, Nicola. "Gene therapy for inborn errors of liver metabolism: progress towards clinical applications." Italian journal of pediatrics vol. 34,1 (2008): 2. doi: https://doi.org/10.1186/1824-7288-34-2
Brunetti-Pierri N. Gene therapy for inborn errors of liver metabolism: progress towards clinical applications. Ital J Pediatr. 2008 Nov 18;34(1):2. doi: 10.1186/1824-7288-34-2. PMID: 19490653; PMCID: PMC2603013.
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Ital J Pediatr. 2008 Nov 18;34(1):2. doi: 10.1186/1824-7288-34-2.

Gene therapy for inborn errors of liver metabolism: progress towards clinical applications.

Italian journal of pediatrics

Nicola Brunetti-Pierri

Affiliations

  1. Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA. [email protected].

PMID: 19490653 PMCID: PMC2603013 DOI: 10.1186/1824-7288-34-2

Abstract

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 transfer technologies. This article summarizes the gene transfer strategies being investigated, the pre-clinical data, and the available early clinical results for inborn errors of liver metabolism.

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