Genomic approaches to identifying targets for treating β hemoglobinopathies

BMC Medical Genomics

Table 2 Approaches to inducing HbF

Method	Drug or locus	Features	References
Pharmacologic	Hydroxyurea^a	Regulatory approval, inexpensive, ineffective in severe β thalassemia, inconsistent response	[141]
	HQK-1001 (sodium 2,2-dimethylbutyrate)	HbF increase of 5-21 % in HbE-β⁰ thalassemia	[142]
	Decitabine	? Epigenetic modification, possible oral route	[143]
	Pomalidomide	Immunomodulatory agent	[144]
	Scriptaid	? Epigenetic modification	[145]
	SAHA	? Epigenetic modification	[146]
Non-pharmacologic
	BCL11A	See text	See text
	MYB	See text	See text
	Direct repeats (DRED) repressors	Point mutations cause HPFH, forced expression increases HbF in sickle mice	[147, 148]
	LCR/HBG promoters	See text	See text

^aHydroxyurea is the sole agent with widespread regulatory approval. Decitabine, scriptaid and SAHA are histone deacetylase inhibitors and other drugs of this general class have also been associated with HbF induction

ISSN: 1755-8794