Human Molecular Genetics, Vol 7, 1655-1658, Copyright © 1998 by Oxford University Press
NF Olivieri and DJ Weatherall
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.
REVIEWS
The therapeutic reactivation of fetal haemoglobin
Division of Haematology/Oncology, Hospital for Sick Children, 555 University Avenue, Toronto, Ontario M5G 1X8, Canada.
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