| Applications
 Hemoglobinopathies
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Sickle
cell disease (SCD) and Cooley's anemia (CA; beta-thalassemia) are
among the most common inherited diseases in humans in the U.S. and
worldwide. ... Despite the fact that the molecular bases of these
so-called beta-chain hemoglobinopathies have been known in many cases
for decades, there are still no universal cures available for these
very serious diseases that are associated with significant morbidity
and in some cases mortality.
Currently, hydroxyurea
and butyrates benefit some patients with SCD or CA, and are
thought to work at least in part through fetal hemoglobin induction,
but they do not benefit all patients, and they have limitations due to
toxicity and route of administration (butyrate).
Actually the National Heart,
Lung, and Blood Institute (NHLBI) and the National Institute of
Diabetes and Digestive and Kidney Diseases (NIDDK) invite applications
for research grants to pursue the unambiguous identification of transactivator
proteins that regulate the expression of fetal globin chains. Both
the validation of existing candidate transactivators, and gene
discovery to identify new ones are encouraged. Increased understanding
of the trans- acting component of developmental stage-specific
hemoglobin isoform switching will facilitate the development of new
approaches to cure beta-chain hemoglobinopathies such as sickle cell
disease and Cooley's anemia.
(From NIH - TRANSACTIVATION
OF FETAL HEMOGLOBIN GENES FOR TREATMENT OF SICKLE CELL DISEASE AND
COOLEY'S ANEMIA
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