Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer
Batshaw, Mark L.
Wilson, James M.
UMass Chan AffiliationsDepartment of Microbiology and Physiology Systems
Division of Hematology/Oncology, Department of Pediatrics
Gene Therapy Center
Document TypeJournal Article
Gene Transfer Techniques
Ornithine Carbamoyltransferase Deficiency Disease
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Nervous System Diseases
MetadataShow full item record
AbstractA 66-year-old woman heterozygous for a mutation in the ornithine transcarbamylase gene (Otc) participated in a phase I gene therapy trial for OTC deficiency. She received an adenovirus (Ad) vector expressing the functional OTC gene by intraportal perfusion. Fourteen years later she developed and subsequently died of hepatocellular carcinoma. A second subject, a 45-year-old woman, enrolled in the same trial presented with colon cancer 15 years later. We sought to investigate a possible association between the development of a tumor and prior adenoviral gene transfer in these two subjects. We developed and validated a sensitive nested polymerase chain reaction assay for recovering recombinant Ad sequences from host tissues. Using this method, we could not detect any Ad vector DNA in either tumor or normal tissue from the two patients. Our results are informative in ruling out the possibility that the adenoviral vector might have contributed to the development of cancer in those two subjects.
SourceHum Gene Ther. 2013 Sep;24(9):814-9. doi: 10.1089/hum.2013.118. Link to article on publisher's website
Permanent Link to this Itemhttp://hdl.handle.net/20.500.14038/30291
Related ResourcesLink to article in PubMed