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

• Հիմնական հեղինակ: Brunetti-Pierri Nicola (Հեղինակ)
• Ձևաչափ: Գիրք
• Հրապարակվել է: BMC, 2008-11-01T00:00:00Z.
• Խորագրեր: article
• Առցանց հասանելիություն: Connect to this object online.

<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...