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Op-Ed: Huntington’s key protein mhTT — Best way for CRISPR to prove what it can do?

Break this bastard of a disease, CRISPR. You CAN do it. It’s just a matter of when.

Neuroscience -Tomography. — © AFP

Huntington’s Disease is a hideous progressive disease. One of the primary causes is a mutant protein called mhTT, which is a corrupted essential protein in spinal fluid. mhTT replaces the proper protein, a protein called whTT, in neurons.

Another related issue includes “protease degradation”. Protease is a type of compound that breaks up proteins. The degradation of protease inevitably causes a train wreck, notably the inability to manage and eliminate waste protein materials.

A global context for a serious health condition

Of the many horrible diseases in this world which truly deserve extermination, Huntington’s Disease is right up there with the very worst of them.  Huntington’s Disease affects millions of people worldwide. An estimated 2.71 people per 100,000 are statistically likely to have this condition. That’s over 20 million people worldwide.

Huntington’s Disease is a truly super-high-maintenance, high-stress, condition. The progression can take years or decades. Neurological effects may include seizures, high levels of pain, and perhaps worse, ongoing trauma. Other symptoms may include necrosis, and apoptosis, aka cell death.

The disease has a long, infamous, history. The most famous person to get this condition was Woody Guthrie, the legendary American folk singer. Guthrie also helped greatly to raise the profile of the disease in public awareness and start the modern campaign to end Huntington’s Disease.

CRISPR to the rescue? Makes so much sense.  

As yet, there’s no cure, but there are a lot of people fighting hard for one.  I see these people doing it every day. It’s inspiring, and it’s incredible. They deserve some help. …And there is some real help available.

Now the good news – The mhTT protein is potentially vulnerable. The vulnerability is based on the option of using gene switches to stop production of mhTT and restore production of whTT.  

(In theory, the body can reboot, if it’s not being progressively sabotaged by more mhTT and degraded proteases. This IS theory, but there also may be ways of restarting the natural protein cycles, too.)

Now, there’s a new major player in the mix – CRISPR. This type of condition is truly core business for CRISPR, which can be used to shut down dangerous gene operations at the source. Also very importantly, it can be used to prevent inheritance of such conditions. Huntington’s Disease involves a “cytosine-adenine-guanine (CAG) trinucleotide repeat expansion in the Huntington gene (HTT)”.

Call it a no-brainer, but this CAG repeat sequence doesn’t look anything like insoluble. Can a CAG repeat sequence be totally inscrutable? It’s a genetic typo, not some incomprehensible mystic problem. Not simple, no. There’s also the issue of getting neural functions and related essentials up and running again. …But nothing like impossible. The target is clearly identified.

In 2018, CRISPR first began to look at Huntington’s Disease. The link includes a clear picture of the possible problems. Point being – This monster of a condition is a primary target for CRISPR, and a place for CRISPR to make major progress.

This is a super-high value line of research for CRISPR. The potential human benefits are invaluable, and so is the science. There’s another possible huge, gigantic upside here – If this gene expression can be reversed, the same process could be used as a template for other horrible conditions. Alzheimer’s and Parkinson’s also involve proteins, like protein plaques, for example.

The range and quality of information CRISPR could get through managing this damn protein could be the template for future cures for so many things. To borrow a quote:

“Not because it’s easy, but because it’s hard.”

And to paraphrase Woody Guthrie (I don’t think he’d mind):

“Build me a world without Huntington’s Disease”.

Break this bastard of a disease, CRISPR. You CAN do it. It’s just a matter of when.

Written By

Editor-at-Large based in Sydney, Australia.

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