Gene therapy for inborn errors of liver metabolism: progress towards clinical applications
<p>Abstract</p> <p>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 th...
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| Format: | Article |
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2008-11-01
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| Series: | Italian Journal of Pediatrics |
| Online Access: | http://www.ijponline.net/content/34/1/2 |
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doaj-9a4c1541424d41a5b1062a7e5c5b3cbd2020-11-25T02:28:21ZengBMCItalian Journal of Pediatrics1720-84241824-72882008-11-01341210.1186/1824-7288-34-2Gene therapy for inborn errors of liver metabolism: progress towards clinical applicationsBrunetti-Pierri Nicola<p>Abstract</p> <p>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.</p> http://www.ijponline.net/content/34/1/2 |
| collection |
DOAJ |
| language |
English |
| format |
Article |
| sources |
DOAJ |
| author |
Brunetti-Pierri Nicola |
| spellingShingle |
Brunetti-Pierri Nicola Gene therapy for inborn errors of liver metabolism: progress towards clinical applications Italian Journal of Pediatrics |
| author_facet |
Brunetti-Pierri Nicola |
| author_sort |
Brunetti-Pierri Nicola |
| title |
Gene therapy for inborn errors of liver metabolism: progress towards clinical applications |
| title_short |
Gene therapy for inborn errors of liver metabolism: progress towards clinical applications |
| title_full |
Gene therapy for inborn errors of liver metabolism: progress towards clinical applications |
| title_fullStr |
Gene therapy for inborn errors of liver metabolism: progress towards clinical applications |
| title_full_unstemmed |
Gene therapy for inborn errors of liver metabolism: progress towards clinical applications |
| title_sort |
gene therapy for inborn errors of liver metabolism: progress towards clinical applications |
| publisher |
BMC |
| series |
Italian Journal of Pediatrics |
| issn |
1720-8424 1824-7288 |
| publishDate |
2008-11-01 |
| description |
<p>Abstract</p> <p>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.</p> |
| url |
http://www.ijponline.net/content/34/1/2 |
| work_keys_str_mv |
AT brunettipierrinicola genetherapyforinbornerrorsoflivermetabolismprogresstowardsclinicalapplications |
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