Changing the world, one gene at a time

A very-very simple introduction to gene editing

Imagine you're building a house. First, you need an architect to design the blueprint. That’s what DNA does. It plans out the construction as it tells your body how to grow, develop, function, etc. But the blueprint isn't enough on its own - you need someone to actually build the house. That's where mRNA comes in - it's like the builder who acts as a messenger, carrying the instructions from the DNA to the ribosomes. And ribosomes? They are the construction workers who read the instructions and assemble the building blocks which are the amino acids. After arranging the amino acids in the required sequence, we finally have a protein that is now ready to carry out the commands of the DNA.

Disclaimer - I realize the actual process is far more dynamic and complex but my nonscientific brain is just going to stick to this parallel.

So Gene editing?

Gene editing is when you can basically find and edit the architect’s layout. It’s a "find and replace/delete" tool for DNA. This can be done in a few different ways, but one of the most promising is CRISPR-Cas9. This technology has garnered significant attention in the scientific community, so much so that in 2020, the Nobel Prize in Chemistry was awarded to American scientist Jennifer A. Doudna and French scientist Emmanuelle Charpentier, for discovering the same.

CRISPR-Cas9 is a tool that allows scientists to precisely cut and edit DNA. Scientists first identify the specific region of DNA that they want to edit. They then design a small RNA molecule called the guide RNA (gRNA), which is like a "molecular address" that tells a specialized enzyme called Cas9 where to cut the DNA. Once the Cas9 enzyme is guided to the right spot by the gRNA, it cuts the DNA in a very specific spot, kind of like a pair of molecular scissors.

Once the DNA is cut, the cell's natural repair mechanisms kick in, and the DNA can be repaired with potential edits that the scientist intended. This is where the gene editing magic happens.

So how does playing around with the blueprint help?

1. Treating genetic diseases: Gene editing could help to cure or treat genetic diseases by correcting the genetic mutations that cause them. For example, sickle cell anemia is a genetic disorder that causes red blood cells to become misshapen, leading to various complications. Gene editing could potentially correct the underlying genetic mutation and cure the disease. Gene editing could also help to prevent the transmission of inherited diseases from parents to their children.

2. Agricultural improvements: Gene editing could help to produce crops that are more resistant to pests and disease, require fewer pesticides and herbicides, and have a longer shelf life. This could help to increase food productivity. (Have written a bit on the future of food here). Could potentially make agriculture more sustainable.

3. Creating new therapies: Gene editing could lead to the development of new therapies for a wide range of diseases, including cancer, HIV, and Alzheimer's disease. By targeting specific genes and altering their function, scientists could potentially develop more effective treatments.

4. Preserving endangered species: Gene editing could be used to help preserve endangered species by modifying the genes that cause diseases or genetic disorders. This could help to prevent the extinction of these species and preserve biodiversity.

But ofcourse, as Spiderman has taught us “With great power comes great responsibility”.

While gene editing has many potential benefits, it also raises ethical questions about how far we should go in manipulating the genetic makeup of living organisms.

1. One of the biggest concerns is the possibility of unintended consequences. Because genes are interconnected, altering one gene could have unforeseen effects on other genes and even entire ecosystems.

2. Another concern is the potential for gene editing to be used for unethical purposes, such as creating "designer babies" or enhancing certain traits deemed desirable by society.

3. There is also the risk of inequality, where only the wealthy have access to gene editing technology, leading to a widening gap between the genetically "haves" and "have-nots".

4. Finally, there are concerns about the long-term effects of gene editing on future generations, as changes made to DNA can be passed down to offspring and potentially alter the course of human evolution.

Okay, any relevance to investors?

Well duh! If we talking about changing the world and “evolution” it’s only natural that the financial markets will care. The obvious applications in healthcare are for everyone to see. It will potentially cure currently incurable diseases.

Moreover, the intersection of gene editing and AI could lead to major breakthroughs in the field. AI could assist in solving the complex problem of protein folding, which has been a major roadblock in developing new drugs and therapies.

Remember, how I touched on, amino acids being the raw materials that are used to create a protein? Turns out that the sequence of these amino acids is critical. The sequence determines how the protein folds into a specific shape, which is critical for its proper function. However, predicting the exact structure a protein will fold into is a complex and difficult problem.

While there are limited amino acids commonly found in proteins, the number of possible combinations and arrangements is enormous. Even small proteins can have hundreds of amino acids. Furthermore, the process of protein folding is not entirely deterministic, as it is influenced by many different factors, including temperature, pH, and the presence of other molecules. This means that even proteins with the same amino acid sequence can fold into different shapes under different conditions. Well shit!

This is where AI can come in handy, as researchers can analyze massive amounts of data to try to better understand how a protein will fold. Easier said than done ofcourse. So yep, a lot of second and third-order consequences for investors to think about.

There is no doubt that gene editing has the potential to revolutionize the field of medicine and agriculture, however, as I said earlier, with great power comes great responsibility!

I am currently reading “The Genesis Machine: Our Quest to Rewrite Life in the Age of Synthetic Biology” by Amy Webb, Andrew Hessel. May potentially interest you, if you enjoyed reading this article.