Gene Editing Technique Corrects Sickle Cell Disease in Mice

937956

Sumary of Gene Editing Technique Corrects Sickle Cell Disease in Mice:

  • The investigators reported using genetic-based editing on mice to convert a disease-causing hemoglobin gene to a benign variant that would enable healthy blood cell production..
  • The root of SCD is two mutated copies of the hemoglobin gene, HBB, which cause red blood cells to transform from a circular disc into a sickle shape..
  • Jude Children’s Research Hospital used a molecular technology called base editing to directly convert a single letter of the disease-causing DNA into a harmless genetic variant of HBB in human blood-producing cells and in a mouse model of SCD..
  • “We were able to correct the disease-causing variant in both cell and animal models using a customized base editor, without requiring double-stranded DNA breaks or inserting new segments of DNA into the genome,”.
  • explained co-senior author David Liu, director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute, a Harvard professor, and a Howard Hughes Medical Institute investigator..
  • “In particular, we combined expertise in protein engineering, base editing and red blood cell biology to create a novel approach for treating and possibly curing sickle cell disease,”…

Want to know more click here go to source.

From -
Close
Generic selectors
Exact matches only
Search in title
Search in content

By continuing to use the site, you agree to the use of cookies. 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