Direct gene transfer with DNA-liposome complexes in melanoma: expression, biologic activity, and lack of toxicity in humans.

• DOI: 10.1073/pnas.90.23.11307
• Publication Date: 2006-05-31T12:36:22Z
• Authors: G J Nabel; E G Nabel; Z Y Yang; B A Fox; G E Plautz; X Gao; L Huang; S Shu; D Gordon; A E Chang
• Keywords: Naked DNA

Direct gene transfer offers the potential to introduce DNA encoding therapeutic proteins treat human disease. Previously, in humans has been achieved by a cell-mediated ex vivo approach which cells from blood or tissue of patients are genetically modified laboratory and subsequently returned patient. To determine feasibility safety directly transferring genes into humans, clinical study was performed. The foreign major histocompatibility complex protein, HLA-B7, introduced HLA-B7-negative with advanced melanoma injection DNA-liposome complexes an effort demonstrate transfer, document recombinant expression, toxicity this therapy. Six courses treatment were completed without complications five stage IV melanoma. Plasmid detected within biopsies treated tumor nodules 3-7 days after but not found serum at any time using polymerase chain reaction. Recombinant HLA-B7 protein demonstrated biopsy all immunochemistry, immune responses autologous tumors could be detected. No antibodies One patient regression injected on two independent treatments, accompanied distant sites. These studies feasibility, safety, direct humans.