Gene Therapy

Major themes within the Gene Therapy Program at Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta include:

Hemophilia A

The translation of high-expression fVIII basic discoveries

  • Recombinant viral vectors incroporating high expression elements can be used as a gene therapy/cure for hemophilia A
  • High-expression elements can be utilized to decrease the cost of recombinant fVIII production
    • Approximately 70 percent of patients with hemophilia A are not treated—a reduction in the cost of goods will increase fVIII product access


Pioneering an approach to treat cancer using novel immunotherapies, termed Drug Resistance Immunotherapy (DRI)

  • Lab research is testing if immunocompetent cells can be genetically engineered to withstand the toxic effects of chemotherapy, and if so, whether the genetic modification can allow for the combined use of chemotherapy and cell-based immunotherapy, which may result in increased survival compared to individual chemotherapy or immunotherapy treatments

Basic Researchers

Recent Publications

  • Doering CB, Archer D, Spencer HT “”Delivery of nucleic acid therapeutics by genetically engineered hematopoietic stem cells”. Adv Drug Deliv Rev. 2010;62(12):1204-12.
  • Dasgupta A, McCarty D, Spencer HT. Engineered drug-resistant immunocompetent cells enhance tumor cell killing during a chemotherapy challenge. Biochem Biophys Res Commun. 2010;391:170-5.
  • Taylor J, Rohatgi  P, Doyle D, Spencer HT, Azizi, B, “Characterization of a Molecular Switch System that Regulates Gene Expression in Mammalian Cells Through a Small Molecular”  BMC Biotechnol. 2010.  [Epub ahead of print.] PMID: 20167077 [PubMed - indexed for MEDLINE]PMCID: PMC2831033

Research Collaborations