Sunday, 8 April 2018

Designer Proteins Could Help Gene Therapy


Gene therapy, or the idea of introducing new genes into the body to treat injuries or illnesses, is still in its infancy, but human genome editing could potentially provide treatment options for previously untreatable conditions. Instead of relying on currently existing proteins, scientists have started experimenting with designer proteins — customized lab designed cells that can complete a certain task.

Guest post by Megan Ray Nichols

What could these designer proteins mean for the future of gene therapy?

Playing With Designer Proteins

Gene therapy normally takes place in a lab, but the future of this practice might rely on computer power instead of genetic samples. Designer proteins are created on a computer and can be modified and adjusted as needed before created in a physical setting.

This isn’t a new practice. In 2000, Stanford University started their Folding@Home program which allowed users to lend their processing power to a protein folding program. Over the last 18 years, the program has helped make breakthroughs in cancer research and infectious and neurological diseases — all using donated processing power from home computers, phones and gaming consoles.

In 2006, Rosetta created the FoldIt game which allowed users to actively modify and fold digital proteins rather than allowing the program to passively do it for them. This later evolved into Cyrus Biotech, which created a commercial cloud platform for projects like FoldIt. This game is still active and updated to this day.

Designer Gene Therapy

The human body contains more than 20,000 different kinds of proteins, so it can be difficult to know how modifying one will change the body — even though we’ve studied them for more than 200 years. That’s where designer gene therapy comes in — scientists can play with an infinite number of permutations without potentially harming a test subject by introducing a designer gene or protein into their body.

These genes, once tested, could potentially have a variety of benefits, from correcting autoimmune disorders like celiac disease, to fighting the flu. The flu virus already utilizes a protein to fuel an infection — the ‘key’ protein to open a door in the cell, allowing it to grow and thrive. By introducing a protein that disables the protein key in the flu virus, gene therapy could effectively nullify the flu’s greatest tool — its ability to spread and infect new cells.

Folded Protein Nanomachines


These folded proteins aren’t just made from cells — some scientists utilize them to create folded protein nanocomputers. These microscopic cellular computers can perform a number of different calculations simultaneously, making them capable of solving problems very quickly. They may not be as powerful as quantum computers, but they may provide some organic processing power that will be vital in the future.

It’s entirely possible that these folded protein nanomachines will be ready for use sometime in the next 10 years.

This might sound like science fiction, but it’s quickly becoming science fact — which is the best kind of fact, if we're honest. There is also the possibility that these sorts of designer genes could be used for evil, a la Gattica, the movie where genetic perfection determines your place in society. Time will tell what these discoveries hold for us in the future.

It might be a while before you can pop down to your local pharmacy for some anti-flu gene therapy, but the last 20 years of protein folding programs have started to shape the way we look at gene therapy and human genome manipulation. It might not be long before we can modify our own genomes to improve health, stop disease and help us become our best selves.

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