Tag Archives: gene therapy
New Treatment for Sickle Cell Disease
Written by Neil Kurtzman | 16th May 2024Sickle Cell Disease is a disorder caused by a single mutation that results in the production of Hemoglobin S. It is an autosomal dominant disease as it is not on a sex chromosome. If both parents possess the abnormal gene and each pass a copy to his/her offspring the child has sickle cell disease. Thus,…
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Gene Therapy for Sickle Cell Disease
Written by Neil Kurtzman | 4th March 2017Sickle 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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