Table 2.
Genes delivered in preclinical and clinical studies
| Gene | Delivery system | Vector | Study | Cartilage defects | Evaluations | References |
|---|---|---|---|---|---|---|
| TG-C | Retrovirus | A phase I dose-escalating clinical trial | Knee advanced OA | Safety and biological activity | NCT00599248-2007 [94] | |
| Retrovirus | A dose-escalating, single-center, phase I clinical trial | Knee degenerative arthritis | Safety and biological efficacy | NCT02341391-2007 | ||
| Retrovirus | A multicenter, single-blind, phase II A clinical trial | Knee degenerative arthritis | Efficacy and safety | NCT02341378-2009 | ||
| Retrovirus | A double-blind, randomized, parallel-group, phase II study | Knee OA grade III | Efficacy and safety | NCT01221441-2010 [95] | ||
| Retrovirus | A placebo controlled, single-blind, randomized, multicenter phase IIB study | Knee degenerative arthritis | Efficacy and safety | NCT01671072-2011 | ||
| Retrovirus | A single-blind, randomized, parallel-group, multicenter phase II study | Knee degenerative arthritis | Efficacy and Safety | NCT01825811-2012 | ||
| Retrovirus | A placebo controlled, double-blind, randomized, parallel group, multicenter phase III study | Knee degenerative arthritis | Efficacy and Safety | NCT02072070-2013 | ||
| TGF-β | Mix (3:1) of unmodified chondrocytes+TG-C | Retrovirus | Ex vivo | Partial cartilage defect or full-thickness knee cartilage defects | Safety, efficacy, toxicity, biodistribution | [81] |
| IL1ra | Direct injection | scrAAV | A phase I study | Knee moderate OA | Local and systemic safety | NCT02790723-2016 |
| FBs | Adenovirus or scAAV | In vitro and ex vivo | Naïve knees or with IL-1β-induced arthritis | Transgenic expression and persistence, infiltrating leukocytes | [60] | |
| Direct injection | scAAV | In vivo | MIA-induced OA | Safety and biodistribution | [97] | |
| TNFR:Fc | Direct injection | rAAV | A double-blind, randomized, parallel group, phase 1 dose escalation study | Inflammatory arthritis | Safety and tolerability | NCT00617032-2004 [98] |
| rAAV | A double-blind, randomized, parallel group, phase I/II study | Inflammatory arthritis | Safety | NCT00126724-2005 | ||
| IFN-β | Direct injection | rAAV | A non-randomized, parallel-group, open-label, pahe I study | RA | Safety | NCT02727764-2016 |
| FLS (in vitro); direct injection (in vivo) | rAAV | In vitro and in vivo | Collagen-induced arthritis | Biodistribution | [99] | |
| Direct injection | rAAV | In vivo | Collagen-induced arthritis | Safety, biodistribution, and efficacy | [100] |