Tuesday, November 22, 2022

The Progression of Gene Therapy Intervention for Sickle Cell Disease


    Sickle cell disease affects thousands of people throughout the globe and is recognized to be prevalent in African populations. Early clinical trials centered around gene therapy for sickle cell disease have been researched by geneticists since the 1990s.The intent of gene therapy for sickle cell disease is to correct genetically modified dysfunctional hemoglobin that leads to the development of structurally altered red blood cells that block blood vessels and lead to complications in organ function. The ultimate goal of research trials centered around gene therapy intervention for sickle cell disease aimed to stimulate the body’s ability to produce fetal hemoglobin by insertion of microRNA. Through insertion of the microRNA into the body, the dysfunctional protein known to alter red blood cell structure was corrected and fetal hemoglobin was successfully produced.

From the research and clinical trials around microRNAs potential to alleviate symptoms associated with sickle cell disease, six participants of the study were shown to have successful outcomes. The research results provide a possible cure to sickle cell disease and hope to many who inherited the illness. 


  1. I discovered in another class that sickle cell anemia is actually an advantage in the prevention of malaria. Since the mutation makes the red blood cells more fragile and likely to burst, they are less likely to spread malaria. Because they are so fragile, I think that the introduction of fetal hemoglobin is a very good idea in that it will prevent further growth of the sickle hemoglobin.

  2. This is very interesting. I know people with sickle cell anemia are in pain a lot of the times because of the abnormal blood cell. I am wondering if this could be used as a therapy for people. This could be a good advancement for treatment of this disease.