Gene editing 101 — Redesign Human Potential
Around 1 in 50 people are affected by a known Single-Gene Disorder, which is kind of insane as people are suffering because of something they can’t control. Almost all Single-Gene Disorder is something you inherited from your parents which means you can’t control it…or can you?
The Human Genome Project
For thousands of years, our genetic makeup was something shrouded in mystery until 1990 when The Human Genome Project started. It was an international scientific research project with the goal to determine the base pairs that make up the human DNA. While mapping all of the genes of the human genome from both a physical and a functional standpoint. It ended in April 2003, and it started an avalanche of new research and discoveries with the biggest one being CRISPR. The clustered regularly interspaced short palindromic repeats, more commonly known as CRISPR, is one of the biggest milestones in the field of Biology and Gene Editing. CRISPR is a powerful tool for editing genomes, allowing researchers to easily alter DNA sequences and modify gene function. To rephrase it we basically have a tool that can edit the very blueprint of life.
So how does CRISPR actually work? Before defining how CRISPR works we need to know how Cas9 works. In nature Cas9 protein is found in bacteria and used when a bacteriophage attacks the bacteria. While attacking the bacteria the bacteriophage injects its DNA into the bacteria. Usually, the bacteria die but when they do survive the bacteria takes the DNA of the bacteriophage and stores it in a DNA archive called CRISPR. Therefore when the next bacteriophage attack happens the bacteria can quickly take an RNA copy of the bacteriophage in order to stop the attack.
Deep Dive: How it works?
There are two main components of CRISPR-Cas9. One is CRISPR which if you break the acronym apart you get Clustered Regularly Interspaced Short Palindromic Repeats which translates roughly to DNA being spaced in intervals. CRISPR is essentially a Gene containing the DNA of past infections. The second main component is the Cas Genes it codes for the Cas Proteins. These proteins are the backbone of the revolution in gene editing as Cas9 can cut any DNA we give it. What makes Cas9 really useful is the fact that it is programmable. Meaning if we think of the old way of genetic engineering like a flip phone then the new CRISPR-Cas9 technology is an iPhone 13.
Why the Hype?
So now that you know how it works why is there so much hype around it? Well, cause it doesn’t just blow the old methods of gene editing out of the water, it basically created its own league. Some of the main reasons it’s revolutionizing are one it’s a more simple protein than older methods. Many old methods were very complex to make, containing many restriction enzymes and other nucleases. Another good reason is that it’s cheaper than the older methods, but even though it’s cheaper doesn’t mean it’s worse than the old methods. As CRISPR-Cas9 is actually more precise than older methods so even though it’s cheaper it still worth it. Lastly, as I said above Cas9 is a homing missile that we can point to any location we want it to land. With the older methods though it wasn’t like that you had limits but now the only limit is our imagination.
Applications of CRISPR-Cas9
The future applications of gene editing are countless but a couple of the big ones, in my opinion, are personalized medicines as of today we use the same medication to treat everyone which is crazy because all of us are different from each other genetically. So with personalized medicine, we can provide treatments that are more effective. Second, it can help with new researches in early humans, extinct creatures, and may even aid in the reanimation of dinosaurs. The third application of CRISPR-Cas9 is the end of genetic diseases as we know it. Ever since the dawn of humans, we had to deal with diseases and now we are advancing into an era where we could possibly eradicate genetic diseases altogether.
