Experiments With Viruses Create Path To Potential Huntington’s Cure

If ever you were in doubt as to what scientists are doing with their free time, fear not they are trying to make the world a much better place.

And that’s exactly what scientists at Emery State have been attempting to do. Make the world a better place by developing a potential cure for Huntington’s disease.

The team there used a process called CRISPR, which involved manipulating genes. In order to partially treat mice who had been infected with the condition.

Huntington’s

For those of you who aren’t familiar with Huntington’s, it’s a horrible degenerative condition. It causes brain cells to break down. Normally this starts to affect people once they reach adulthood and their brain starts to make proteins that are “toxic” to brain cells.

The study, which was published in the Journal of Clinical Investigation was performed on mice who had been genetically engineered to have the condition. When the mice became 9 months old their bodies started to exhibit symptoms associated with Huntington’s such as impaired movement.

Genetic manipulation

Here’s the clever part the team was able to use CRISPR to alter the genes of the animals. This resulted in mice that, whilst they weren’t cured, showed much improvement.

The method of delivery for this therapy was in itself quite interesting. The code was injected into the mouse’s brain in the form of a virus. This technique is known as using an adeno-associated virus, or AAV. It was specifically targeted on the part of the mouse’s brain that controls movement.

Senior author Professor Xiao-Jiang Li commented on the scope of the findings suggesting that they create pathways for new treatments. But not just Huntington but other inherited neurodegenerative diseases as well. She was cautious though stating that further testing was required to make sure safety and long-term effects are taken into consideration.

Caution

Of course, this could be huge news but as with any breakthrough of this nature, it’s important for scientists to tread carefully. Yes, it’s safe to say that using CRISPR could eventually give doctors an amazing weapon in their arsenal that could have all sorts of positive ramifications.

But employing the technology before it is fully understood could be catastrophic. Just look at the thalidomide scandal! That resulted in a huge number of babies being born with a tragic birth defect which ended up misshaping their arms.

I mean lets for a second assume that scientists did start using this technology on human’s right away. We know which gene to target but we don’t yet know if targeting that gene could have a knock-on effect. We also don’t know if changing that gene could have ramifications further down the road. Although the signs are encouraging it is far too soon for us to start dreaming that using CRISPR is going to be a magical cure. Once again Li commented emphasising the need for further testing. While studies like these give hope it is important that we never rush ahead with using them.

Huntington’s

The future

So consequently there are no plans in order to start using this therapy in order to fight conditions such as Huntington’s.

Hopefully, that will start to happen in the future. In the meantime what is encouraging is that using CRISPR scientists also believe that they can cure certain forms of cancer. That research is far more advanced and human trials are underway in both the US and China.

I guess there are those that would point to this sort of research and highlight the controversial nature of it. Is it a step too far? Another example of a man playing god? Or if it can save and improve lives is it worth it?

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