Looking from the Outside at the Magic of CRISPR Will Enhance Effective Gene Therapies

From futirism.com

The First “Cell-Free” CRISPR Tech Is Here To Personalize Cancer Treatment

 

…..“When you’re working with CRISPR inside a cell, you’re kind of working in a black box where you can’t really observe the gears of the machinery that are doing these amazing things,” Eric Kmiec, lead researcher and director of the Gene Editing Institute, said in a press release. “You can see the results, the edits to the genes, but not necessarily how you got there, which is important for ensuring that CRISPR can be safely used to treat patients.”

According to the researchers, theirs is the first CRISPR tool that can do this, and it could be an improvement over CRISPR-Cas9 in several respects.

First: it lets researchers see what CRISPR is actually doing. As Kmiec mentioned, this is important so that scientists can make sure the treatment will be safe for patients.

Second, it’s a quicker way to conduct diagnostic tests. The mutations that cause cancer aren’t the same in every patient. A doctor could use CRISPR-Cpf1 to determine the specific mutation causing the disease in a patient more quickly than with CRISPR-Cas9, according to Kmiec. This, in turn, could help that doctor determine the best treatment option for their patient.

Third, the specific cuts made by Cpf1 could be more useful than those made by Cas9. When Cas9 makes a cut, it leaves “blunt ends” on the gene. No big deal if cutting is all a researcher wants to do, but the blunt end isn’t great for attaching to a new bit of genetic code. Cpf1, on the other hand, leaves “sticky ends” that make it easier for a researcher to insert new DNA.

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