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CRISPR Gene Editing Tools May Not Work On Most Humans

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What is gene editing?

CRISPR has been introduced as the end-all-and-be-all solution for human problems since its inception. Genes could now be edited to get rid of problems that humans are facing.

A new study now shows that expectations may have to be tempered quite a bit.

60 Percent Of The Time, It Works Every Time

A new study shows that the CRISPR Cas9 gene editing system may not work on most humans. The problem was that between 65 percent to 79 percent of humans had antibodies that would fight the Cas9 proteins. This means that most people have an immune system defenses against what's supposed to cut genetic diseases out of genes.

This is because the enzyme in the Cas9 protein is derived from the bacteria Staphylococcus aureus and Streptococcus pyogenes, which commonly infect humans. Being common infections means that they have evolved along with humans, which means that the immune system has developed a response to both strains of bacteria.

The study has yet to be peer reviewed but can signal trouble for the companies that are investing in the gene editing system. Human trials are supposed to be around the corner, but it's still not clear if the Food and Drug Administration will allow the companies to carry out the tests.

Workarounds

There are alternatives to Cas9. Scientists are already studying other types of CRISPR that use different proteins. Besides the use of different proteins, there are also different methods for delivery into the body.

One of the methods involves removing the cells from the body of the patient, fixing the DNA, and then returning it to the body. This would make it possible for there to be little or no CRISPR proteins remaining on the cells for the immune system to fight against.

"Like any new technology, you want to identify potential problems and engineer solutions for them," said Dr. Matthew Porteus, a senior author on the study. "And I think that's where we're at. This is an issue that should be addressed."

This is a stumbling block in the development of the CRISPR technology that has shown promise as a tool in the fight against genetic diseases. Something to keep in mind is that the study hasn't been peer reviewed just yet. Clinical trials on humans are set to start this year. In Europe, CRISPR Therapeutics will begin trials to treat beta thalassemia patients.

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