Table 2.
Examples of preclinical studies on RNA viral-based gene therapy and vaccines.
| Viral Vector | Disease | Target | Response | Ref |
|---|---|---|---|---|
| Retroviruses | ||||
| RRV/Toca511+5-FC | Glioma | CD | Prolonged survival in mice | [59] |
| GRV | X-CGD | SINfes.gp91s | Protection against A. fumigatus | [60] |
| RV | Cancer | NK cells | Support for GMP grade production | [61] |
| COL7A1 | RDEB | Collagen VII | Reversed RDEB in mice | [62] |
| RV | XP | XPC | Skin regeneration in mouse model | [63] |
| Lentiviruses | ||||
| HIV-1 | PD | RNAi | Down-regulation of α-synuclein | [65] |
| HIV-1 | PD | GAD67 | Normalized neuronal activity | [66] |
| HIV-1 | AD | RNAi | Reduction in neurodegeneration | [67] |
| HIV-1 | AD | siRNA | Reduction in tau phosphorylation | [68] |
| HIV-1 | SCI | AQP4-RNAi | Accelerated motor function | [69] |
| HIV-1 | PKD | PKLR | Corrected hematological phenotype | [70] |
| HIV-1 | β-thalassemia | β-globin | Therapeutic efficacy, no toxicity | [71] |
| HIV (BB305) | SCID-X1 | IL2RG | Restoration of T, B, and NK cell counts | [72] |
| HIV-CAR-T | AML | CD123 | Rapid elimination of leukemia in mice | [73] |
| HIV-CAR-T | Ovarian CA | TAA | Potential ovarian cancer treatment | [74] |
| HIV-CAR-T | SHIV | CD46-CD4 | Protection against SHIV | [75] |
| Cal-1 anti-HIV | SHIV | Cal-1 | Safe integration | [76] |
| Alphaviruses | ||||
| SFV | Lung CA | EGFP | Prolonged survival | [79] |
| SFV | Glioma | IL-12 | Tumor regression, prolonged survival | [80] |
| SFV | Glioma | miRNAs | Tumor targeting, prolonged survival | [81] |
| M1 | Liver CA | oncolytic M1 | Tumor growth inhibition | [82] |
| VEE | EBOV | EBOV NP | Protection against Ebola virus | [83] |
| VEE | EBOV | EBOV GP, NP | Protection against Ebola virus | [84] |
| SFV | EBOV | EBOVGP, VP40 | Neutralizing antibodies | [85] |
| SFV | HIV | Env | Humoral response | [87] |
| SFV | HIV | Env/Gag/Pol | Antigen-specific immune response | [88] |
| VEE | Influenza | HA | Protection against influenza in chicken | [89] |
| SFV | Influenza | HA, NP | Protection against influenza in mice | [90] |
| VEE | Influenza | HA | Protection against influenza in mice | [91] |
| VEE | Lassa | GP | Protection against Lassa in mice | [92] |
| Flaviviruses | ||||
| KUN | Colon CA | G-CSF | Tumor regression | [93] |
| KUN | Melanoma | G-CSF | Tumor regression | [93] |
| Zika virus | Glioblastoma | Glioma cells | Specific killing of GSCs | [94] |
| KUN | SIV | SIV gag-pol | Protection against SIV | [95] |
| Rhabdoviruses | ||||
| VSV | EBOV | EBOV-GP | Protection against EBOV in macaques | [96] |
| VSV | EBOV | EBOV-GP | Protection against EBOV in primates | [97] |
| VSV | MARV | MARV-GP | Protection against EBOV in primates | [97] |
| VSV | Melanoma | VSV-GP | Prolonged survival in mice | [99] |
| VSV + ruxilitinib | Ovarian CA | VSV-GP | Oncolytic activity | [100] |
| VSV | Prostate CA | VSV-GP | Long-term remission in mice | [101] |
| Measles viruses | ||||
| MV | Dengue | Dengue DV2 | Neutralizing antibodies in mice | [102] |
| MV | Dengue | Dengue DV1-4 | Protection against Dengue in mice | [103] |
| MV | HBV | HBsAg | Humoral response in mice, primates | [104] |
| MV | Brain CA | SLAM, EGFR | Tumor regression in mice | [105] |
| MV | Breast CA | CEA | Prolonged survival in mice | [106] |
| MV | Lung CA | SLAM | Tumor suppression in mice | [107] |
| MV | Pancreatic CA | SLAM | Tumor suppression in mice | [108] |
| MV | Prostate CA | CEA | Prolonged survival in mice | [109] |
| NDV | ||||
| NDV TS09-C | NDV | NDV-GFP | Protection against NDV in chicken | [52] |
| NDV | Neuroblastoma | NDV 73-T | Complete tumor regression in mice | [110] |
| NDV-F | Colon CA | IL-2 | Long-term remission in mice | [111] |
| NDV LaSota | Melanoma | IL-15 | Suppression of tumor growth in mice | [112] |
| NDV D90 | Lung CA | NDV D90, GFP | Suppression of tumor growth in mice | [113] |
| NDV | Melanoma | IL-2 + TRAIL | Prolonged survival in mice | [114] |
| NDV | HCC | IL-2 + TRAIL | Prolonged survival in mice | [114] |
| NDV Anhinga | HCC | TRAIL | Cure and protection against re-challenges | [115] |
| VSV-NDV | HCC | VSV-NDF | Prolonged survival in mice | [116] |
| Picornaviruses | ||||
| CVB3 | IN | FGF2 | Protection against ischemic necrosis | [117] |
| CAV21 | Melanoma | ICAM1, DAF | Tumor regression, also in metastases | [118] |
| CAV21 | Breast CA | ICAM1, DAF | Improved tumor regression | [119] |
| CAV21 + DH | Breast CA | ICAM1. DAF1 | Tumor regression, dose dependence | [120] |
| CAV21, EV1 | Prostate CA | DAFv | Superior tumor regression | [121] |
| CVB3 | Coxsackievirus | CVB3-IFN-γ | Protection against Coxsackievirus | [55] |
5-FC, 5-fluorocytosine; AD, Alzheimer’s disease; AML, acute myeloid leukemia; AQP4, Aquaporin-4; CA, carcinoma; CAR-T, chimeric antigen receptor T cell; CAV21, Coxsackievirus A21; CEA, carcinoembryonic antigen; CD, cytosine deaminase; CVB3, Coxsackievirus B3; DAF, decay-accelerating factor; DH, doxorubicin hydrochloride; EBOV, Ebola virus; G-CSF, Granulocyte-Colony Stimulating Factor; GP, glycoprotein; GRV, gammaretrovirus; EGFR, epidermal growth factor receptor; FGF2, fibroblast growth factor-2; GSCs, glioblastoma stem cells; HA, hemagglutinin; HBV, hepatitis B virus; HBsAg, hepatitis B surface antigen; HCC, hepatocellular carcinoma; HIV, human immunodeficiency virus; ICAM-1, intercellular adhesion molecule-1; IL2RG, interleukin-2 receptor γ gene; IL, interleukin; IN, ischemic necrosis; KUN, Kunjin virus; MARB, Marburg virus; miRNAs, micro-RNAs; MV, measles virus; NDV, Newcastle disease virus; NP, nucleoprotein; PD, Parkinson’s disease; PKD, pyruvate kinase deficiency; PKLR, pyruvate kinase isoenzymes L/R; RDEB, Recessive Dystrophic Epidermolysis Bullosa; RNAi, RNA interference; RV, retroviral vector; RRV, retroviral replicating vector; SCI, spinal cord injury; SCID, severe combined immunodeficiency; SHIV, Simian/Human Immunodeficiency Virus; SFV, Semliki Forest virus; siRNA, small interfering RNA; SLAM, signaling lymphocyte activation molecule; TAA, tumor associated antigen; TRAIL, tumor necrosis factor-related apoptosis inducing ligand; VEE, Venezuelan equine encephalitis virus; VSV, vesicular stomatitis virus; VSV-GP, VSV pseudotyped with lymphocytic choriomeningitis virus glycoprotein; X-CGD, X-linked chronic granulomatous disease; XP, Xeroderma pigmentosium.