Tag Archives: hemoglobin s

Gene Therapy for Sickle Cell Disease

Sickle Cell Disease is a disorder of hemoglobin production secondary to an alteration in the hemoglobin gene on chromosome 11.  Specifically, there is a single amino acid substitution in “adult” β A -globin (Glu6Val) stemming from a single base substitution (A→T) in the first exon of the human β A -globin gene. The abnormal nucleotide…

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