Abbreviations: IFNγ, interferon gamma; IL-2, interleukin 2; TK, thymidine kinase (sometimes used with bromodeoxyuridine); HSV-TK, herpes
simplex thymidine kinase, often coupled with gancyclovir treatment; BRCA-1, breast cancer 1, early onset; PSA, puromycin-sensitive aminopeptidase;
CEA, carcinoembryonic antigen; GM-CSF, granulocyte-macrophage colony stimulating factor; MDR-1, multi-drug resistance protein 1 (used to insert
chemotherapy-resistance genes into the hematopoetic lineage); CD80, protein involved in T-cell activation; CD, cytosine deaminase; TNF, tumor necrosis
Definitions of phases in a clinical trial: Phase I usually fewer than 100 healthy volunteers, primarily to gauge adverse reactions, and to determine optimal
dose and best route of administration. Phase II are generally pilot efficacy studies usually involving 200500 volunteers randomly assigned to control and
study groups. Phase II will test for immunogenicity in the case of vaccines, and duration of expression and amelioration of symptoms for gene therapy. Note
that none of these trials have proceeded beyond Phase II, and most are in Phase I.
Source: Wiley Journal of Gene Medicine/Clinical Trial Database at: http://www.wiley.co.uk/genetherapy/clinical/.
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Nonviral Vectors for Gene Therapy
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Viruses as Vectors for Heterologous
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