Publication: Fetal globin gene inducers: Novel agents and new potential
Issued Date
2010-01-01
Resource Type
ISSN
17496632
00778923
00778923
Other identifier(s)
2-s2.0-77955906235
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Mahidol University
Rights Holder(s)
SCOPUS
Bibliographic Citation
Annals of the New York Academy of Sciences. Vol.1202, (2010), 158-164
Suggested Citation
Susan P. Perrine, Serguei A. Castaneda, David H.K. Chui, Douglas V. Faller, Ronald J. Berenson, Noppadol Siritanaratku, Suthat Fucharoen Fetal globin gene inducers: Novel agents and new potential. Annals of the New York Academy of Sciences. Vol.1202, (2010), 158-164. doi:10.1111/j.1749-6632.2010.05593.x Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/28568
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Title
Fetal globin gene inducers: Novel agents and new potential
Abstract
Inducing expression of endogenous fetal globin (γ-globin) gene expression to 60-70% of alpha globin synthesis produces β-thalassemia trait globin synthetic ratios and can reduce anemia to a mild level. Several classes of therapeutics have induced γ-globin expression in beta-thalassemia patients and subsequently raised total hemoglobin levels, demonstrating proof-of-concept of the approach. Butyrate treatment eliminated transfusion requirements in formerly transfusion-dependent patients with treatment for as long as seven years. However, prior generation inducers were not readily applicable for widespread use. Currently, a novel oral dual-action therapeutic, sodium 2,2-dimethylbutyrate, is in clinical trials, an oral decitabine formulation is under development, and agents with complementary mechanisms of action can be applied in combined regimens. Identification of three major genetic trait loci which modulate clinical severity provides avenues for developing tailored regimens. These refinements offer renewed potential to apply fetal globin induction as a treatment approach in patient-friendly regimens that can be used worldwide. © 2010 New York Academy of Sciences.