TY - JOUR
T1 - Histone deacetylase inhibitors and hemoglobin F induction in β-thalassemia
AU - Migliaccio, Anna Rita
AU - Rotili, Dante
AU - Nebbioso, Angela
AU - Atweh, George
AU - Mai, Antonello
PY - 2008
Y1 - 2008
N2 - Epigenomic modifiers, such as histone deacetylase inhibitors, are compounds that regulate gene expression by interfering with the enzymatic machinery that maintains the proper chromatin structure of the nucleus. These compounds are at the forefront of novel therapeutic agents for the treatment of several diseases including cancer and genetic disorders such as β-thalassemia and sickle cell disease. Here we review the current understanding of the mechanism of action of epigenomic modifiers in the treatment of β-thalassemia and sickle cell anemia. We also discuss how the lessons learned from the study of the effects of these compounds on the β-globin locus, one of the best characterized regions of the human genome, might contribute to the understanding of the mechanism of action of these same compounds in cancer, where the specific regions of the genome that are responsible for the pathophysiology of the disease are often poorly defined.
AB - Epigenomic modifiers, such as histone deacetylase inhibitors, are compounds that regulate gene expression by interfering with the enzymatic machinery that maintains the proper chromatin structure of the nucleus. These compounds are at the forefront of novel therapeutic agents for the treatment of several diseases including cancer and genetic disorders such as β-thalassemia and sickle cell disease. Here we review the current understanding of the mechanism of action of epigenomic modifiers in the treatment of β-thalassemia and sickle cell anemia. We also discuss how the lessons learned from the study of the effects of these compounds on the β-globin locus, one of the best characterized regions of the human genome, might contribute to the understanding of the mechanism of action of these same compounds in cancer, where the specific regions of the genome that are responsible for the pathophysiology of the disease are often poorly defined.
KW - Hemoglobin F induction
KW - Histone deacetylase inhibitors
KW - β-Globin locus
KW - β-Thalassemia
UR - http://www.scopus.com/inward/record.url?scp=48349094044&partnerID=8YFLogxK
U2 - 10.1016/j.biocel.2008.04.024
DO - 10.1016/j.biocel.2008.04.024
M3 - Short survey
C2 - 18617435
AN - SCOPUS:48349094044
SN - 1357-2725
VL - 40
SP - 2341
EP - 2347
JO - International Journal of Biochemistry and Cell Biology
JF - International Journal of Biochemistry and Cell Biology
IS - 11
ER -