Charting Pediatrics podkast

A Solution to Sickle Cell: New Gene Editing Therapy Could Lead to a Pain Free Life

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You’ve likely heard of CRISPR, the comprehensive gene editing technology that emerged in the 2010s and has since revolutionized biological research. Additionally, the medical community has long been using the concept of gene therapy. What’s new is that both approaches have now successfully been harnessed for the treatment of human disease. In December 2023, the FDA approved two therapies for the treatment of sickle cell disease; one utilizing CRISPR technology and the other using a gene transfer therapy approach. These therapies now have the opportunity to change the life course of this terrible disease.  

Our guest for this episode is Christopher McKinney, MD, who specializes in pediatric hematology here at Children’s Hospital Colorado. Dr. McKinney has been the hospital’s site principal investigator in some of the clinical trials that are contributing to these new sickle cell therapies, and he is an assistant professor of pediatrics at the University of Colorado School of Medicine.  

Some highlights from this episode include: 

  • How these treatments work for diseases like sickle cell 
  • What we know about the effectiveness of these therapies thus far 
  • Important side effects and risks to take into consideration 
  • Regardless of FDA approval, what barriers are in place 
  • Powerful patient examples 

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