The Power to Change DNA: CRISPR-Cas9 and Sickle Cell Disease

History was made on December 5, 2020, when the New England Journal of Medicine published a paper titled CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. How did we get to this point in 2020 and what has been accomplished in the intervening five years?

Jennifer Domm is a hematologist oncologist who has been working with a courageous group of more than 50 sickle cell sufferers who have volunteered to participate in clinical trials that are improving treatment options for a debilitating and painful inherited disease. Domm will share the story of how CRISPR-Cas9 gene editing developed and describe how the technology has helped her treat an intractable, inherited, and painful blood disorder that affects some 100,000 Americans.

Cas9 is a protein that works like a scalpel to cut DNA at a particular location. Like the hand that holds a scalpel, guide RNA then targets a specific location for a change or repair to the segment of DNA that needs inactivation or modification. In this way genetic mutations can be corrected, and new treatments for diseases developed. The promise of CRISPR-Cas9 extends beyond medical treatment. It can also be used to study genetic functions or to develop pest and disease resistant agricultural products. When it delivers a patient from the traumas of pain and frequent hospitalizations to a “normal” life it seems nothing short of miraculous.

Even after this treatment gained FDA approval for cell-based gene therapies for the treatment of sickle cell disease in 2023, there remain many barriers to commercial access. Domm will present and answer questions on the science and data behind this therapy that promises some relief to patients who experience debilitating pain and other serious complications that accompany sickle cell disease.

This Joseph Priestley Society talk is coproduced by the American Chemical Society as part of its ACS Webinars series.