Table 3.

Preclinical studies and clinical trials on gene- and cell therapy for hemophilia

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

LVV, lentiviral vector; AAV, adeno-associated vector; FIX, factor IX.