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2.

Cellular targets of ER intrabodies

ER Intrabodies inhibiting oncogenic proteins
Target Target function Cellular targets Performance References
Human IL-2 receptor Signal transduction Jurkat T cells In vitro studies with intrabody plasmid [29]
Human IL-2 receptor Signal transduction T-cell leukaemia cell line, HTLV-1 transformed cell lines and primary human T cells In vitro studies with tetracycline responsible intrabody plasmid and HIV-1-based lentivirus vector [30, 31]
ErbB-2 Signal transduction NIH/3T3 cells expressing human erbB-2 and mammary carcinoma cells In vitro studies with murine leukaemia virus vector [32, 33]
ErbB-2 Signal transduction Human breast cancer and ovarian carcinoma cell lines In vitro studies with recombinant adenovirus and intrabody plasmid [34, 35]
ErbB-2 Signal transduction Human ovarian carcinoma cells transplanted in SCID mice In vivo studies with recombinant adenovirus [36]
ErbB-2 Signal transduction Human ovarian cancer cells Phase I study with ovarian cancer patients performed with recombinant adenovirus [37]
ErbB-2 Signal transduction Human erbB-2-positive tumour cell lines In vitro study with intrabody plasmid containing a high affinity anti-erbB-2 scFv fragment [38]
ErbB-2/androgen receptor Signal transduction Human prostate cancer tumour cell line In vitro studies with murine leukaemia virus vector [39]
EGFR Signal transduction Human tumour cell lines overexpressing EGFR In vitro studies with murine leukaemia virus vector [40]
VEGFR-2/KDR Signal transduction Porcine aortic endothelial cells overexpressing humanVEGFR-2 In vitro studies with intrabody plasmid [41]
VEGFR-2/KDR Signal transduction HUVECs In vitro studies with recombinant adenovirus [42]
Tie-2 Signal transduction Mouse model with Kaposi's sarcoma and human colon carcinoma xenografts In vivostudies with adenovirus-delivered anti-Tie-2 intrabody [43]
VEGFR-2/KDR-Tie-2 Signal transduction Mouse model with human melanoma xenografts In vivo studies with adenovirus-delivered anti-VEGFR-2-Tie-2 intradiabody [44, 45]
Human α folate receptor Signal transduction Human ovarian carcinoma cell lines In vitro studies with intrabody plasmid [46]
Cathepsin L Endopeptidase Human melanoma cell lines In vitro studies with intrabody plasmid [81]
Metalloproteinase MMP-2 and MMP-9 Degradation of collagen IV Human lung carcinoma cell line In vitro studies with intrabody plasmid [82]
ER intrabodies targeting HIV-1 gp160/gp120 and HIV-1 co-receptors
Target Target function Cellular targets Performance References
HIV-1 gp120 Viral coat protein Human lymphocytes and CD4+ T cells from HIV-positive patients In vitro studies with recombinant adeno-associated virus and murine leukaemia virus vector [47, 48]
HIV-1 gp160 Viral coat protein CD4+ human T cells (Jurkat) In vitro studies with intrabody plasmid [49]
CCR5 HIV-1 co-receptor CCR5+/CD4+ human lymphocyte cell line In vitro studies with murine leukaemia virus vector [50]
CCR5 HIV-1 co-receptor CD34+ fetal liver stem cells In vitro studies with HIV-1-derived self-inactivating lentivirus vector and in vivo studies with NOD/SCID mice transduced with the intrabody expressing CD34+ fetal liver stem cells [51]
CCR5 HIV-1 co-receptor Human monocyte-derived macrophages and microglia cells from human fetal brain tissue In vitro studies with Tag-deleted SV40-derived vector and hammerhead CCR5 specific ribozyme [52]
CXCR4 HIV-1 co-receptor CD4+Human T lymphocytic cell line and HeLa-CD4/ βgal-CCR5 cells In vitro studies with murine leukemia virus and SV40-based virus vector [53]
CXCR4 HIV-1 co-receptor Primary human brain microvascular endothelial cells and post-mitotic differentiated human neurons In vitro studies with HIV-1-based lentivirus vector [54]
ER intrabodies targeting other viruses as HIV-1
Target Target function Cellular targets Performance References
Hepatitis C virus core protein Viral core protein Human hepatocellular carcinoma cell line and HEK 293 cells In vitro studies with intrabody plasmid [83]
Maedi-visna virus gp46 protein Envelope glycoprotein Sheep choroid plexus cell line In vitro studies with the soluble scFv fragment, intracellular ER expression of the antibody is planned [84]
Root-knot nematode Meloidogyne incognita Nematoide in fection Tobacco leaf protoplasts In vitro studies with intrabody plasmid [85]
ER intrabodies abrogating antigen presenting molecules
Target Target function Cellular targets Performance References
MHC I Antigen presentation Primary rat keratinocytes In vitro studies with intrabody plasmids [55]
MHC I Antigen presentation Human CD4+ Jurkat T-cell line, human primary keratinocytes and several cell lines of divergent tissue sources In vitro studies with intrabody plasmid and recombinant adenovirus [56]
MHC I Antigen presentation Human umbilical vein endothelial cells In vitro studies with recombinant adenovirus [57]
α1,3-Galactosyl-transferase Glycosylation Pig epithelial kidney cells In vitro studies with intrabody plasmid [87]
ER intrabodies targeting integrins
Target Target function Cellular target Performance References
αV integrin Mediation of cell-cell and cellmatrix interactions Saos-2 human osteosarcoma cell line and WM-266-4 melanoma cell line In vitro studies with intrabody plasmid [58, 59]
αV integrin Mediation of cell-cell and cell-matrix interactions Human metastatic melanoma cell lines and xenograft melanoma SCID mouse model In vitro and in vivo studies with recombinant adenovirus [60]
ER Intrabodies targeting proteins involved in Alzheimer's and Prion disease
Target Target function Cellular target Performance References
Human β-amy-loid precursor protein (APP) Transmembrane protein involved in the pathogenesis of Alzheimer's disease HEK 293 cells transient transfected with cDNA of human APP In vitro studies with intrabody plasmid [28]
Cellular prion protein Pathogenesis of Prion diseases Nerve growth factor-differentiated PC12 cells infected with a suspension of mouse brains from scrapie-affected mice and scrapie mouse model In vitro and in vivo studies with PC 12 cells stably expressing the intrabody [61, 62]