Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
SE Raper, N Chirmule, FS Lee, NA Wivel… - Molecular genetics and …, 2003 - Elsevier
We report the death of an 18-year-old male with partial ornithine transcarbmaylase (OTC)
deficiency who participated in a pilot (safety) study of gene therapy. The vector used for this
trial was based on human adenovirus type 5, deleted in E1 and E4, and contained human
OTC cDNA. It was infused into the right hepatic artery at a dose of 6× 1011particles/kg.
Approximately 18h. following gene transfer the subject was noted to have altered mental
status and jaundice—clinical signs not seen in any of the first 17 subjects in this study …
deficiency who participated in a pilot (safety) study of gene therapy. The vector used for this
trial was based on human adenovirus type 5, deleted in E1 and E4, and contained human
OTC cDNA. It was infused into the right hepatic artery at a dose of 6× 1011particles/kg.
Approximately 18h. following gene transfer the subject was noted to have altered mental
status and jaundice—clinical signs not seen in any of the first 17 subjects in this study …