First U.S. Patients Treated With CRISPR As Human Gene-Editing Trials Get Underway

LisaCovington

First U.S. Patients Treated With CRISPR As Human Gene-Editing Trials Get Underway

The powerful gene-editing technique called CRISPR has been in the news a lot. And not all the news has been good: A Chinese scientist stunned the world last year when he announced he had used CRISPR to create genetically modified babies.

But scientists have long hoped CRISPR — a technology that allows scientists to make very precise modifications to DNA — could eventually help cure many diseases. And now scientists are taking tangible first steps to make that dream a reality.

For example, NPR has learned that a U.S. CRISPR study that had been approved for cancer at the University of Pennsylvania in Philadelphia has finally started. A university spokesman on Monday confirmed for the first time that two patients had been treated using CRISPR.

One patient had multiple myeloma, and one had sarcoma. Both had relapsed after undergoing standard treatment.

The revelation comes as several other human trials of CRISPR are starting or are set to start in the U.S., Canada and Europe to test CRISPR's efficacy in treating various diseases.

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NickOttens

@MarthaDeevy, @abarsouk, @Elena_Milova, you may be interested in this. Any thoughts?

abarsouk

CRISPR offers a tempting fix to many simple and deadly genetic mutations, but we must remember the legacy of gene therapy (at the very same UPenn) some 20 years ago, when clinical trial Jesse Geisenger succumbed to rashly planned and executed new technology. There are known drawbacks to CRISPR, like the risk of off target nicking and cancer promotion, that must be addressed before large human trials

arshimehboob

Targeted hormone replacement therapies, if initiated carefully in the appropriate clinical population, hormone replacement therapies in men and women may prevent and reverse muscle and bone loss and functional declines and perhaps promote healthy aging and longevity.