Thursday, April 7, 2022

Gene Therapies for Sickle Cell Disease Come with Hope and Challenges


Sickle cell disease is a genetic condition in which red blood cells (RBCs) are unable to flow properly throughout the body due to their misshapen form. This disease affects millions across the globe and over 100,000 here in the United States. While a change in a key protein is to blame for the resulting effects, geneticists have been puzzled for many years in terms of finding a cure.

Thanks to advances in modern technology, Pediatricians like Erica Esrick can talk about existing sickle cell treatments and ongoing clinical trials that show positive advancements towards finding a cure through gene therapy. “Gene therapies seek to manipulate the very information of life by replacing, inactivating, or fixing missing or broken genes — and so curing patients. Despite gene therapy’s challenges, many researchers believe sickle cell is a good target because the molecular pathways are well understood and straightforward. What’s more, every copy of the gene doesn’t need to be mended to have an effect” (Garcia de Jesus, 2022). Fetal hemoglobin, a healthier form of hemoglobin, is being chosen to be the target of gene therapy trials.

Preliminary data released in January 2021 showed that the treatment helped six sickle cell patients make fetal hemoglobin. “Scientists are testing other ways to tackle sickle cell via gene therapy, too. A biotechnology company called bluebird bio is testing an approach that delivers a functional copy of the HBB gene to patients. Another team is preparing to begin a trial that will edit that gene directly using CRISPR/Cas9” (Garcia de Jesus, 2022).

As someone who is headed into the healthcare field, it makes me happy that I will have new treatments and cures to offer my patients who suffer from this condition. I believe that in the years to come genetic treatments and therapies will start to play a larger role in the healthcare industry. This case is only the start of something revolutionary. 

In addition, I include a related article on sickle cell disease and its potential treatments. 

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