CRISPR could cure the disease, without changing genes


CRISPR is our genetic superweapon. Derived from the cellular machinery of bacteria, scientists have already started using it to fight everything from cancer to HIV. This works mainly by cutting small pieces of genetic code and replacing it with special sequences designed to correct errors in our genetic code. While it is quite sweet on its own, it can still cure more mundane diseases without cutting or splicing genes at all.

In a new article published in the journal Cell, researchers at the Salk Institute propose using CRISPR to not edit genes, but to turn them off and turn them on instead.

This comes after some questions that have been raised about the safety of CRISPR. There are some studies that suggest that the technique sometimes makes mistakes, which causes unpredictable (and possibly dangerous) changes in the genetic code. So far, the alarms have not been triggered, and CRISPR has had many successes, but when we are using the code that makes us, it is important to be safe.

"The recent revolution in engineered gene editing technologies has opened a new door for targeted precision medicine," study co-authors Fumiyuki Hatanaka and Hsin-Kai Liao told Gizmodo. "However, generating double-strand breaks has the risk of causing unwanted permanent mutations, which is an obstacle to using CRISPR to treat humans."

This new technique takes advantage of an evolving understanding of how genomes work. For some time, much emphasis was placed on the unprocessed genetic code. That is, the letters that represent base pairs that your cells read and use to make proteins. More recently, however, studies have focused on the effects of the more complex pieces of DNA as a whole molecule: how closely it is injured, whether genes are activated or deactivated at a given point and why.

This is particularly useful, because, as noted above, genes can be flipped as switches. It is not necessary that you cut them, necessarily. And cutting is risky, as with any machine, the more steps you enter, the more things can go wrong. When CRISPR goes to work, it also traverses both strands of DNA, which means that you must be absolutely sure that you are re-entering the correct code, or you could end up with random gibberish at a critical point. And that could mean that you die. Or have cancer OR get cancer and die.

The Hatanaka and Liao method removes the clipping portion of the clipping, and has already been used successfully in mice with diabetes, kidney disease and muscular dystrophy. All that was needed was to activate or deactivate certain codes as necessary, and in a short time, the test subjects improved dramatically.

It's a beginning, but a promising one that could generate dividends in the future. The whole field of genome editing is progressing rapidly, and there is great potential in many of the techniques and tools that researchers are now developing to cure many of humanity's most pernicious diseases and even stop aging or design super-humans totally new. In fact, we live in interesting times.

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