Turns Out Our Bodies May Be Putting Up Roadblocks to Gene-Editing

Here’s some humbling news this morning. I came across an article that points out we may not be quite as close to wide use of CRISPR-Cas9 as we sort of think we are.

A recently released scientific paper “that is yet to be peer-reviewed” says that CRISPR-Cas9 may be running into some roadblocks inside our very own bodies.

This very complex sentence from the paper pretty much says it all, “The presence of pre-existing adaptive immune responses in humans to either Cas9 homolog may hinder the safe and efficacious use of the Cas9/gRNA system to treat disease, and may even result in significant toxicity to patients.

Let me explain.

This impacts the part of the technology that “cuts” the DNA. Since the Cas9 side of CRISPR-Cas9 generally uses “protein…derived from the bacteria Staphylococcus aureus (S. aureus) and Streptococcus pyogenes (S. pyogenes)” to cut DNA, the authors of the paper wanted to know if there would be an immune response from the body since these bacteria commonly infect human beings.

More specifically, the researchers “looked for the presence of pre-existing adaptive immune responses to their respective Cas9 homologs, SaCas9 (S. aureus homolog of Cas9) and SpCas9 (S. pyogenes homolog of Cas9).”

Ultimately the researchers from Stanford University did find incidence of immune response (you can check out the paper to see how) in the test tube.

As the article in FireceBiotech points out, “In essence, humans are infected by these bacteria all the time, and thus many of us will have an immune response to deal with them. So, when using the Cas9 protein, which is where the so-called “snipping” tool of CRISPR-Cas9 comes from, many patients may not benefit as their immune systems will fight back.”

So what does this mean? Well it sort of remains to be seen, but I think it points out that gene editing may not quite as close to getting into the clinic as we all seem to think hope it is. We know that it’s still a bright, shining new technology, but it must be refined before we start shooting it into patients.

There’s a difference between HOPE and REALITY. It is more than appropriate to HOPE for great things to become a REALITY, but until we can touch and feel a REALITY where gene editing can have an impact in people with chronic health conditions, we have to be weary of fantastic headlines.

Now I know FDA recently approved gene therapies this past year for certain disorders, but we can’t automatically assume this is all coming to us on a timeline that seems convenient for our personal health issues.

As with any sort of scientific breakthrough (or medication development), I stress cautious optimism when looking towards it. It’s up to us to make sure we are as healthy as can be, so that when these options may be viable in 5, 10 or 20 years, we are able to benefit from them!