Would you get CRISPR'd?

Verve, a new biotech company, is developing a one-shot CRISPR gene-editing drug. Verve is employing a new technology known as base editing, which acts like a pencil and eraser, substituting a single letter of DNA for another. The gene Verve seeks to edit, PCSK9, regulates LDL levels in the body. Verve’s strategy for now is to focus on patients suffering from an inherited form of high cholesterol known as familial hypercholesterolemia, or FH, which increases the likelihood of having coronary heart disease at a younger age.

FDA-approved treatments for high cholesterol currently include not only statins but newer injections targeting PCSK9. But these treatments don’t change the bad gene so the patient has to take the drugs repeatedly.

The gene edit is only for somatic (regular body) cells. It won’t be transferred to later generations.


Dangerous diseases caused by a single mutation include cystic fibrosis and hemophilia. The concept of a “gene editing space” in the pharmaceutical arsenal would have been unthinkable before CRISPR was invented. These single-gene mutations are low-hanging fruit since the diseases are deadly and the problem is caused by a single mutation.

It’s a little creepy to think of having one’s genes edited. Once done, there’s no going back.

Would you get CRISPR’d once it becomes available?



Dangerous diseases caused by a single mutation include cystic fibrosis and hemophilia.

Would you get CRISPR’d once it becomes available?

If I had either of these diseases, you bet.



It’s a little creepy to think of having one’s genes edited. Once done, there’s no going back.

If they can edit once, why can’t they edit again?


It’s a little creepy to think of having one’s genes edited

These thaerapes come with a price…


The U.S. Food and Drug Administration on Wednesday approved bluebird bio’s (BLUE.O) gene therapy for patients with a rare disorder requiring regular blood transfusions, and the drugmaker priced it at a record $2.8 million.

Not exactly a direct CRISPR modification, transduction of autologous CD34+ cells using lentiglobin viral vector BB305.
You’re still modifying your genetics code and with a fairly high risk for blood cancer.

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As I approach my 37th 35th birthday, I would be willing to be a guinea pig and take one for the team especially if it could benefit me too.

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It’s the technology I gave a heads-up on in this post.


The WSJ article is behind a paywall but from the minimal amount that’s displayed, it almost implies that this technology would be a useful alternative for folk who’re non-compliant with their current therapy…and possibly folk who require lipid lowering meds because they’re also non compliant with the lifestyle choices? From the reading I’ve done since Peter Attia’s article, this technology…along with PCSK9 inhibitors (that are currently available)…are intended for those folk with inherited, not acquired dyslipidemia. A club that I’ve recently become a reluctant member of…much to my surprise.

Based on such preliminary data and number of subjects, I wouldn’t be standing online for this…but hope it’s the wave of the future for any future generations who I may have gifted with the dodgy genes.


So, tomorrow I have my CT angiogram as a prelude to flow analysis via the technology I mentioned yesterday…


My visit with an intervention cardiologist on Monday was a take on the adage “It ain’t what you don’t know that gets you into trouble…” and I came away aware that what I used to know just ain’t so any longer. I’d imagined that all manner of aggressive diagnostics and interventions were on the docket for me and had no idea that enlightened medical management (with judicious use of statins and PCSK9 inhibitors) can achieve therapeutic LDL levels of 50 and below that can be demonstrated to cause a regression of atherosclerotic plaques rather than just prevent new ones.

I guess stent manufacture isn’t going to be a growth industry moving forward…although given the increase in obesity and its consequences increasing in children, that might be overly optimistic.

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Something like that is already verified as working for people with sickle cell anemia. Saw that on (60 Minutes?) 1-2 years ago.