The therapeutic reactivation of fetal haemoglobin.
Unusually high levels of fetal haemoglobin production can ameliorate sickle cell disease and beta thalassaemia. Although efforts directed at the pharmacological stimulation of fetal haemoglobin as an approach to managing these conditions have met with limited success, there is wide variation in individual responses. Whether this reflects the particular mutations that underlie these conditions or other genetic factors remains to be determined, as does the ideal combination of agents to achieve this end. These results are encouraging, however, in particular in view of the recent demonstration that other monogenic diseases, Duchenne muscular dystrophy, for example, might be amenable to the same therapeutic strategy.
Olivieri
N F
NF
Division of Haematology/Oncology, Hospital for Sick Children, 555 University Avenue, Toronto, Ontario M5G 1X8, Canada.
Weatherall
D J
DJ
D000755:Anemia, Sickle Cell / Q000097:blood / Q000235:genetics / Q000628:therapy*; D000986:Antisickling Agents / Q000627:therapeutic use; D002087:Butyrates / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D005319:Fetal Hemoglobin / Q000096:biosynthesis* / Q000235:genetics; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use; D015854:Up-Regulation / Q000187:drug effects; D017086:beta-Thalassemia / Q000097:blood / Q000235:genetics / Q000628:therapy*
D000986:Antisickling Agents; D002087:Butyrates; D005319:Fetal Hemoglobin; D006918:Hydroxyurea