Gene Therapy for Hyperbilirubinemia
The term “gene therapy” is used to designate a process in which viruses are used to carry specific genetic information into a cell, thereby transducing the cells’ function. In\the case of the Crigler–Najjar syndrome, and the Gunn rat, the absence of the bilirubin-conjugating enzyme glucuronosyl transferase can be corrected in cells infected by the modified viral vectors. Overall the numbers of inborn errors of metabolism susceptible to treatment with gene therapy, as well as the potential treatment of other types of disorders will be highly significant.
KeywordsGene Therapy Enzyme Replacement Therapy Partial Hepatectomy Unconjugated Bilirubin Unconjugated Hyperbilirubinemia
- Toietta, G., et al. (2005) Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector. Proc. Natl. Acad. Sci. 102:984–989Google Scholar