Table 3. New therapeutic approaches in... | American Society of Hematology

Table 3.

New therapeutic approaches in preclinical development for thalassemias

Drug	Mechanisms	Material	Reference
Minihepcidins	Hepcidin upregulation	Th3/+ mice	34,35
TMPRSS6-LRx	TMPRSS6 disruption	Th3/+ mice, monkeys	36
Exogenous transferrin	TfR1 downregulation	Th3/+ mice	37
ERFE downregulation	Hepcidin upregulation	Th3/+ mice	33,36
IOX1	Selective silencing of α-globin	Erythroid cultures	39

Drug	Mechanisms	Material	Reference
Minihepcidins	Hepcidin upregulation	Th3/+ mice	34,35
TMPRSS6-LRx	TMPRSS6 disruption	Th3/+ mice, monkeys	36
Exogenous transferrin	TfR1 downregulation	Th3/+ mice	37
ERFE downregulation	Hepcidin upregulation	Th3/+ mice	33,36
IOX1	Selective silencing of α-globin	Erythroid cultures	39