Monday, April 30, 2018

Using CRISPR to Edit Genes

I have written about CRISPR gene editing several times over the years. I can see  it someday removing the defective CAG repeats in Kennedy's Disease.

Last night, there was a CBS program ’60 Minutes’ featuring CRISPR. The segment was well done. It broke down the actual process to understandable bites of information.

If you missed the program, you can read about it and watch most of it on 60 Minutes Overtime.

I was impressed to learn 2,300 labs across the globe are currently experimenting with CRISPR. And, they have used it to correct a genetic disorder that causes blindness (mouse models only). There is the first U.S. clinical trial using CRISPR for patients with a certain cancer. I also found it interesting that scientists have to consider what could be any unexpected results of correcting the defective DNA.

Below are excerpts of the CBS article:

“Scientists are excited about using CRISPR to treat genetic disease, but the devil's in the details.

Take, for example, treating cystic fibrosis. Scientists know that the hereditary disease is caused by the misspelling of a particular gene. Genes are made up of DNA, which is composed of chemical bases abbreviated by the letters A, T, G, and C. A genetic mutation that causes a disease like cystic fibrosis reads like a typo in the genetic instructions. If scientists can identify the typo, they can program CRISPR to try to correct it with the right sequence.

This week on 60 Minutes, correspondent Bill Whitaker reports on the gene-editing tool. Feng Zhang, a molecular biologist at the Broad Institute of MIT and Harvard, who has been working on CRISPR for seven years, explains how it works.” ...

"You can understand why I want to say, 'It's amazing,' and you also have to be careful and every single cure is going to take a lot of people working together to deliver it," Lander tells Whitaker in the video above. "But you couldn't have imagined such a cure. It wasn't even on your to-do list before CRISPR." ...

"Preventing disease, I think, is a little more complicated because it depends on how we're trying to prevent the disease. Some of them may have undesirable side effects that we don't fully understand," he explains in the clip above.”

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