Table 3 Preclinical studies and clinical trials on gene- and cell therapy for hemophilia
From: Advanced therapies for the treatment of hemophilia: future perspectives
Authors [Reference] | Vector or target (tissue) cells | Coagulation factor expressed | Expression level (%) |
|---|---|---|---|
Gene therapy (Preclinical studies) | |||
Jeon et al. Ref.[35] | LVV | VIII | 1-5 |
Brown et al. Ref.[34] | LVV | IX | 10 |
Ramezani et al. Ref.[36] | LVV | VIII | <40 |
Matsui. Ref.[37] | LVV | VIII | <40 |
Montgomery and Shi. Ref.[40] | LVV | VIII | <40 |
Gene therapy (Clinical trials) | |||
Nathwani et al. Ref.[38] | AAV (Immunosuppressive therapy) | IX | 2-11 |
Buchlis et al. Ref.[39] | AAV | IX | FIX RNA expression and AAV DNA persistence (<1% FIX) |
Cell therapy (Preclinical studies) | |||
Aronovich et al. Ref.[43] | Embryonic day 42 spleen tissue | VIII | 30-40 |
Follenzi et al. Ref.[44] | Liver sinusoidal endothelial cells | VIII | 14-25 |
Follenzi et al. Ref.[45] | Kupffer cells. Bone marrow-derived mesenchymal stromal cells | VIII | 10-15 |
Xu et al. Ref. [46] | iPSCs from tail-tip fibroblasts and their differentiation into endothelial cells and their precursors | VIII | 8-12 |
Yudav et al. Ref. [47] | Transdifferentiation of iPSC-derived endothelial progenitor cells into hepatocytes | VIII | 20 |