The Nobel prize winning technology that could be bigger than COVID: CRISPR-Cas9 wins the Nobel Prize
A break from COVID
While many of us - myself included - are preoccupied with the worsening pandemic and the upcoming election, there’s some very big non-COVID science news being made.
Earlier this month, Jennifer Doudna and Emmanuelle Charpentier were awarded the Nobel Prize for their work discovering and developing CRISPR-Cas9 genome editing technology.
This technology is a big fucking deal.
A world with developed CRISPR technology will look different than the world we currently live in. This is generational technology with a long-run scientific impact that may actually dwarf our current pandemic.
What is CRISPR-Cas9 technology?
CRISPR is an acronym that stands for “clustered regularly interspaced short palindromic repeats.”
Cas9 stands for CRISPR associated system 9.
Um… English please?
Bascically, CRISPR refers to short DNA sequences that are found in some bacterial genomes that make up a very primitive immune system in these single celled organisms.
When a bacteria is attacked by a virus (called a bacteriophage, which is a virus that affects bacteria), it inserts a piece of the viral DNA into its own DNA.
The bacteria can then identify this foreign DNA - i.e. DNA from a similar invading virus - using the inserted piece of foreign DNA, which is placed adjacent to a CRISPR sequence, sort of as a signaling tool.
The Cas proteins then do the actual work, cutting the foreign DNA, disrupting the invading virus and providing bacterial immunity to a recognized foreign invader.
Doudna and Charpentier recognized that this bacterial immune system could be harnessed to perform targeted DNA editing if primed with the right guide post.
What does this actually means for humans?
When it’s fully developed, CRISPR-Cas9 technology holds the promise of editing our DNA like a Microsoft Word Document.
Being able to perform targeted DNA editing could mean taking a gene variant we inherited that causes disease and swapping it for a variant that doesn’t have the same risk.
This mean the possibility of curing single gene diseases like sickle cell anemia or cystic fibrosis in our lifetimes.
It may also mean a vaccine to prevent heart disease.
Or personalized cancer treatments.
CRISPR-Cas9 technology holds promise to revolutionize every aspect of medical care.
As we learn more about genetics, we recognize that very few human diseases are controlled by just a single gene. Most of the diseases that we know - diabetes, cancer, high blood pressure - have multiple genes that can impact them.
But as we get better at translating the human genome, and as CRISPR technology matures, gene editing may start making its way into every single area of medicine.
The CRISPR revolution has already started
Even though these treatments haven’t been widely deployed yet, we’re starting to see the clinical trials.
Phase 1 trials are already underway to treat inherited amyloidosis. People have already been treated for sickle cell anemia.
And CRISPR isn’t confined just to medicine.
There’s potential for CRISPR to transform agriculture, giving a new meaning to genetically modified foods. China is banking on this technology to provide food for its huge population.
You may even be seeing CRISPR mushrooms that have been engineered to resist browning in a grocery store near you.
And it wouldn’t be an installment of my newsletter without a COVID reference, so you can be sure that there’s discussion of using CRISPR to fight our current pandemic.
So there’s a lot to be excited about.
Just because it’s exciting doesn’t mean it’s ready for prime time.
Editing DNA isn’t the type of technology that we should be deploying widely before we understand the consequences much better than we do now.
I don’t just mean on the ethical front, although there are a huge amount of ethical concerns, and not just in the realm of turning Earth into Gattaca.
I won’t get into the ethics here, but suffice it to say there’s a lot to talk about.
The easier question to pose is about safety.
When you edit DNA, you better be damned sure that you’re doing it 100% precisely before you start injecting that technology into humans. Off target DNA editing is as bad as it sounds, potentially causing cancer or other unexpected biologic effects.
The burden of proof needs to be on the folks who want to deploy this technology to demonstrate safety, rather than on other investigators to determine that it’s unsafe.
The unknown-unknowns of DNA editing raise an almost infinite number of questions.
Perhaps we should be employing the precautionary principle for CRISPR before we just dive in and start widely employing it.
Exercising caution isn’t something that we did before we widely deployed GMO foods into our system, and perhaps that was a mistake.
I expect that the future is going to be filled with applications of CRISPR - for better or for worse.
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