Friday, October 1, 2021

Clinical trails of gene therapy are starting to show improvements for sickle cell anemia


 According to an article in the New York Times, there have been advancements in gene therapy for those suffering from sickle cell anemia. The article discusses the journey of a specific patient named Helen Obando who was the first Am6erican teenager to become sickle cell free from these clinical trails. The technique used as of late to treat sickle cell anemia is a bone marrow transplant from someone who does not have the disease and is a close enough match. However, there are cases in which a match cannot be found (like Obando's). 

    There are three types of clinical trials involving the treatment of the disease using gene therapy. The first, being the trial Obando underwent, used the technique to turn off her adult gene used to form hemoglobin and turn on her fetal one. The reason behind turning on the fetal gene is due to its inability to carry the sickle cell gene whereas the adult gene does. This allows perfectly formed red blood cells to be produced if the trial is successful. The second trial applied by Vortex used CRISPR in order to turn on and off those specific genes. CRISPR is used to target specific genes within the DNA and is a great technique when used to point out the hemoglobin producing gene. The third trial is being produced by Bluebird Bio which uses an inactive virus to give to the patients that held the appropriate hemoglobin gene. Unfortunately, Bluebird Bio had to pause its trial because two of its patients developed cancer.

    The unfortunate truth about sickle cell anemia is that the African American population are primarily born with the disease. Due to this commonality of the disease between African Americans, the progress for finding new methods for a cure have been happening slower than what may occur for non-minority groups. The reasoning behind this is up to opinion, but I think it is worth a conversation to have and that our heath care system may need to take a step back to reevaluate their focus. 


  1. Absolutely, it is imperative that we look inwards and re-assess what goals we have in mind for medical procedures and who gets them. I can only imagine the uses we will have for turning genes on and off when other diseases come into mind. Perhaps a way to regrow organ tissue using fetal genes? I'd love to see more.

  2. The idea of turning on one gene in order to benefit another is a situation where the pros and cons have to be weighed out, and the best decision considering the situation has to be made. I believe the researchers will use information like this to find a cure, or at least something to help sickle cell disease, like you said in the last paragraph. Technology is improving every day.