The therapeutic reactivation of fetal haemoglobin

Unusually high levels of fetal haemoglobin production can ameliorate sicklecell disease and beta thalassaemia. Although efforts directed at thepharmacological stimulation of fetal haemoglobin as an approach to managingthese conditions have met with limited success, there is wide variation inindividual responses. Whether this reflects the particular mutations thatunderlie these conditions or other genetic factors remains to bedetermined, as does the ideal combination of agents to achieve this end.These results are encouraging, however, in particular in view of the recentdemonstration that other monogenic diseases, Duchenne muscular dystrophy,for example, might be amenable to the same therapeutic strategy.