Preferred Label : HSV- thymidine Kinase-m2/hGM-CSF Genes-encoding GEN2;
NCIt synonyms : HSV Thymidine Kinase-m2 and hGM-CSF Genes GEN2; HSV-tk-m2/hGM-CSF Genes GEN2;
NCIt definition : A gene therapy agent composed of a vector expressing a mutated form of the herpes
simplex virus thymidine kinase (HSV-tk) gene and the human cytokine granulocyte-macrophage
colony-stimulating factor (GM-CSF) gene, with potential immunostimulating and antineoplastic
activities. Upon administration, the HSV-thymidine kinase-m2/hGM-CSF genes-encoding
GEN2 selectively infects tumor cells and expresses HSV-tk-m2 and GM-CSF. Subsequent
administration of a synthetic acyclic guanosine analog, such as valganciclovir, allows
the activation of this prodrug by HSV-tk to form ganciclovir. Once phosphorylated
intracellularly, ganciclovir triphosphate competitively inhibits deoxyguanosine triphosphate
(dGTP) incorporation into DNA and inhibits DNA synthesis. This kills the tumor cells
expressing HSV-tk. The release of tumor-associated antigens (TAA) by dying tumor cells
may then stimulate an antitumor cytotoxic T-lymphocyte (CTL) response, directed against
any remaining tumor cells. The expressed GM-CSF enhances antigen presentation, promotes
natural killer (NK) cell-mediated killing and causes a CTL response against tumor
cells, resulting in an immune-mediated tumor cell death.;
Molecule name : GEN-2; GEN 2;
NCI Metathesaurus CUI : CL1642551;
Origin ID : C176770;
UMLS CUI : C5447426;
Semantic type(s)
- concept_is_in_subset
