Sickle Cell Anemia
Sickle cell gene specifically silenced by a single nucleotide polymorphism
Published in Gene Therapy Weekly, June 15th, 2006
"A SNP in the sickle beta-globin gene (ps) leads to sickle cell anemia. Sickling increases sharply with deoxy sickle Hb concentration and decreases with increasing fetal gamma-globin concentration," wrote D.M. Dykxhoorn and colleagues, Harvard University.
"Measures that decrease sickle Hb concentration should have an antisickling effect. RNAi uses small interfering (si)RNAs for sequence-specific gene silencing. A beta(s) siRNA with position 10 of the...
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Source: Gene Therapy Weekly (2006-06-15)
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