Breakthrough Gene-Editing Treatment Helps Child Born With Rare Disorder
Ask students: What is gene therapy? Why was gene therapy used to help K.J. Muldoon? How did CRISPR work?
Doctors announced this week that they have treated a newborn baby with a rare genetic disease using the world’s first personalized gene editing therapy. Geoff Bennett discussed the treatment and its potential with Dr. Peter Marks. He oversaw gene therapy treatment and vaccine safety and approval for the FDA before he left in March.
View the transcript of the story.
Discussion Questions
- Who are the individuals featured in the video?
- Where was K.J. Muldoon being treated?
- What is gene therapy?
- Why was gene therapy used to help K.J. Muldoon?
- How did CRISPR work in the case of K.J. Muldoon?
Focus Questions
- How could the gene therapy discussed in the story be scaled (used) to treat other diseases?
- What are the ethical considerations involved in the cost of treatment of gene therapy?
- To what degree has the Trump administration's cuts to medical research affected the U.S.'s position as a leader in gene therapy, according to Dr. Marks? (Learn more about cuts to medical research here.)
- Media literacy: There has been debate for some years now over the ethics involved in using gene therapy tools like CRISPR. How could you find out more about this topic and if concerns over its use have changed over the years?
Extension Activity
- Learn more about how CRISPR works by reading this Stanford University article featuring scientist Dr. Stanley Qi discussing what CRISPR is, how it works and ethical concerns.
- Take a look at this News Hour story, Why the FDA’s approval of revolutionary sickle cell gene therapy is a ‘big deal.’ Sickle cell disease is a chronic, debilitating condition that affects nearly 100,000 Americans, most of them with African ancestry. Now, the FDA has approved a groundbreaking treatment for it that uses the gene-editing tool CRISPR.
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Republished with permission from PBS News Hour Classroom.
PBS News Hour Classroom helps teachers and students identify the who, what, where and why-it-matters of the major national and international news stories. The site combines the best of News Hour's reliable, trustworthy news program with lesson plans developed specifically for... See More
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