The gene editing tool called CRISPR was recently used for the first time in the United States at the University of Pennsylvania in Philadelphia.

“In a nutshell, CRISPR is a technique that has been developed that allows one to edit DNA in cells,” said Dr. Jill Keeney, professor of biology at Juniata College. In our cells we have chromosomes, with genes on the chromosomes made up of bases...and when we get changes in those, that affects how they function.”

Three patients have been treated as part of the study for the past three months, and the initial report seems promising.

Two of the patients have multiple myeloma, a blood cancer, and the third has a sarcoma, cancer that forms in soft tissue.

The plan is to treat 15 more patients to assess the technique’s effectiveness.

“With these approved trials, if it works, it would be really phenomenal. It’s a powerful technique. If it works, we could fix cells that have these errors. The issue with it is that there have been other gene therapies that have been around for a long time and these techniques always end up having side effects, something we call off target insertions,” said Keeney.

“What has to happen is you have to put in DNA with the right sequence to fix the DNA with the wrong sequence, but when you put it into cell it can go into places you don’t want it to go. The genome is so big it’s difficult to control that.”

This new technique has a level of sophistication other therapies can’t compare with, however.

“CRISPR is far, far more accurate than other therapies. CRISPR can actually fix the problem where it’s at.”

It’s not easy to tell when, if ever, gene editing techniques will become common practice, as there is much that’s unknown.

“It’ll depend on outcome of these trials. It has to be approved first,” said Keeney

“We don’t know how long after that it will take to determine if there are side effects: one year or two year or five years. And we don’t know how permanent the change CRISPR makes would be. There’s no reason to think the changes would not be permanent but we don’t know. It is a new scientific technique.”

Nathan can be reached at


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