• Join Today!

Become a member and connect with:

  • An Active Online Community
  • Articles and Advice on SCD
  • Help Understanding Clinical Trials

‘Natural’ Gene Mutation May Offer Way of Treating Sickle Cell Disease, Study Says


Introducing a “natural” genetic mutation into the blood cells of people with sickle cell and like diseases, using CRISPR gene editing, was seen to restore the production of fetal hemoglobin and offer a way to treat these disorders, researchers report.

Their study was published in the journal Blood, in an article titled “KLF1 drives the expression of fetal hemoglobin in British HPFH.”

Sickle cell disease (SCD) patients have a genetic defect that leads them to produce hemoglobin S, or sickle hemoglobin, instead of normal hemoglobin. This impairs the ability of red blood cells to bind to oxygen molecules in the lungs, and consequently to deliver oxygen to tissues throughout the body.

https://www.onescdvoice.com/wp-content/uploads/2017/11/Mutation.jpg

To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More information

The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.

Close