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graphic representation of CRISPR using birds

CRISPR technology offers hope for sickle-cell patients

A Stanford Medicine article examines CRISPR, the gene-editing technology, and addresses its potential to help with conditions such as sickle-cell disease.

In my office, which communicates findings from Stanford Medicine researchers to the media and the public, we take a lot of pride in accurately communicating science. We scrupulously avoid hype and try to temper expectations – research is, after all, incremental and revolutionary breakthroughs are few and far between.

But, after reading the CRISPR article in the latest issue of Stanford Medicine magazine, I'm struggling to rein in my enthusiasm. CRISPR, as I'm guessing you've heard, is a relatively new technique that allows for researchers to quickly snip out genes and replace them with others.

There are well-founded worries about the dangers and ethical dilemmas involved with using CRISPR technology in humans. But there is also hope.

Hope for patients with genetic diseases like David Williams, a 15-year-old whose life has been shaped by sickle-cell disease. Sickle-cell disease is caused by two defective hemoglobin genes, which produce sickle-shaped red blood cells. Those with the disease suffer from severe pain and a host of complications.

As early as next year, a team of Stanford researchers and clinicians led by Matthew Porteus, MD, PhD, could begin enrolling participants in a clinical trial to fix one of the defective genes in stem cells of patients with sickle-cell disease. Before the trial kicks off, more work is needed to show that stem cells altered with CRISPR can be tested in humans. But, the researchers realize the work has the potential to help many people with sickle-cell disease.

"The repaired stem cells could create enough normal red blood cells for the patient to be symptom-free for life," Porteus says in the article. "That's the ultimate goal."

I've managed to remain composed this far, but just have to say that if their efforts work – wow!

Illustration by Jason Holley

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