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.