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

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 been using gene therapy for quite some time. However, a more recent development is that both approaches have been successfully harnessed to treat human disease. In December 2023, the FDA approved two therapies for treating sickle cell disease: one using CRISPR technology and the other a gene transfer therapy approach. These therapies now have the potential to change the life course of this terrible disease. 

Listen to our pediatric expert discuss gene editing therapy

In this episode, Christopher McKinney, MD, joins us to share his insights on gene editing and his experience as a site principal investigator for some clinical trials that have contributed to these new sickle cell therapies.   

Dr. McKinney specializes in pediatric hematology at Children’s Hospital Colorado and is an assistant professor of pediatrics at the University of Colorado School of Medicine. 

In this episode, our expert discusses:  

• 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 treatment stories