I tend to daydream a lot especially when I was younger. I’d spent countless amounts of time each day wondering, what would I do if I was one day given the option to choose a superpower. What power would I even pick? — I was a huge nerd when it came to DC or Marvel Superheroes 😆.
Would I want to fly like superman or perhaps run at the speed of light? I just loved superheroes and the idea of having cool powers. 12 years ago, I was sure that there was no way such abilities could exist and that it was all fiction.
However, with recent technologies, we could change that. Before we dive into that, I want you to meet this hero who has a really interesting origin story. His name is…
I’m sure you’ve heard of the man of steel, Superman. Superboy is similar to Superman in terms of the abilities they have. He can fly, leap over buildings in a single bound, X-ray vision and basically the whole superman package.
After the death of Superman at the hands of Doomsday (a Kryptonian monster), they wanted to create a clone replacement of Superman. This was done through genetic engineering to fuse the DNA of a human and a Kryptonian.
In short, Superboy was made possible thanks to science and more specifically, genetic engineering. Believe it or not, genome editing technologies have existed in the late 1900s.
In fact, two biochemists named Emmanuelle Charpentier and Jennifer Doudna discovered a groundbreaking gene-editing tool known as CRISPR/Cas 9 in 2009 that completely disrupted the biomedicine field!
Genetically Modified Superhumans
With CRISPR/Cas 9 technology, reaching a world filled with superhumans isn’t totally impossible. The bigger question would be — Should we create superhumans?
There are so many things to consider when it comes to answering this question and it tends to be a huge debate on the ethics side. I think, for now, we’re at a crossroads.
While we can’t predict what the future would look like with gene editing, we can use it for the immediate good. If we can genetically modify superhumans, we can do the same for people who are genetically impaired.
It is roughly estimated that 400 million people worldwide suffer from rare diseases with most of them being genetics-based. For most of these diseases, conventional medicine cannot offer them a cure but only serve to manage its symptoms.
With the recent breakthrough in gene-editing technology, Jennifer Doudna co-founded the company called Intellia Therapeutics. They’re using gene editing to help people with rare genetic, oncological and immunological diseases.
They’re also trying to create a modular gene-editing platform to create various in vivo (within the body) and ex vivo (basically cell therapy outside the body) pipelines.
Essentially, they’re creating CRISPR/Cas 9-based medicines for genetic diseases and cell therapies for various cancers and autoimmune diseases.
Curing Transthyretin Amyloidosis
You’ve probably never heard of this disease and I don’t blame you. It is, after all, a rare disease with good reason. Transthyretin Amyloidosis is also known as ATTR amyloidosis and it’s essentially a transthyretin protein disorder.
It’s a mouthful ( I still can’t say it right 😆) so let’s break that down:
Amyloidosis is a protein disorder. Protein misfolds, binds together and forms structures called fibrils which deposits itself in organs. As you can expect, this can cause serious complications to the affected organs.
Transthyretin is a protein made by the liver that helps transport the thyroid hormone and vitamin A in the blood. combine these two together and you get ATTR amyloidosis.
In ATTR amyloidosis, the protein becomes unstable, breaks apart and deposits itself in the heart and/or nerves. There are two types of ATTR:
- Hereditary ATTR amyloidosis (from abnormal DNA)
- Wild-type ATTR amyloidosis (from ageing)
Intellia Therapeutics is more focused on curing hereditary ATTR amyloidosis which is caused by a DNA mutation that makes the transthyretin protein more unstable causing it to misfold.
In fact, the first patient was dosed with their investigational genome editing treatment for transthyretin amyloidosis (ATTR) in November 2020.
A New Approach to Cas 9 Delivery
CRISPR/Cas 9 is an amazing tool for gene editing but the method of delivering the Cas 9 enzyme is equally as important to reach high gene editing efficiency.
Conventional methods used viral vectors to deliver the Cas 9 enzyme into the body but there were a couple of potential limitations that brought down its overall efficiency:
- It's capable of triggering pre-existing and adaptive immune responses
- Expression from these vectors are sustained, increasing the risk of side effects
Intellia took a new approach and used a non-viral vector to deliver their investigational treatment for ATTR amyloidosis. It’s called the lipid nanoparticle (LNP) and there are several reasons why it’s more efficient.
- Transient & non-integrating Cas 9 expression to limit off-target events
- Capacity to transport large Cas 9 enzyme and 1 or more guide RNA
- Option for repeated dosing is available
- Enables large-scale manufacturing
The combination of Intellia’s lipid nanoparticle managed to achieve more than 70% editing and more than 90% protein knockdown with a single dose of the co-formulated LNP.
The editing and protein knockdown was even consistent for 12 months making it very durable. However, LNP currently only works for liver-based genetic diseases and their program, NTLA-2001 is still undergoing phase 1 of clinical trials at the moment.
Super Powering Humans
Like I’ve mentioned above, we haven’t actually defined where the line will be drawn when it comes to genetically modifying human for enhancement purposes.
In fact, super powering humans could cause a societal backlash if not done in a fair and just manner. If enhancements were sold, we could see greater polarization between the rich and the poor.
If gene editing was democratized and accessible to all, how would that be regulated? What’s stopping me from starting my own Jurassic Park or injecting myself with animal genes to enhance my body?
Although that line is still blurred, we can still superpower humans who have debilitating diseases. To me, that’s the first step to super powering humans the right way.
- Super-powering humans who are born with genetic diseases is the right step in the right direction.
- Intellia therapeutics is solving ATTR amyloidosis, a rare disease with CRISPR/Cas 9 gene editing
- Instead of using viral vectors, Intellia uses lipid nanoparticles which are more efficient
Hello From The Author!
Hey hey! Thank you for reading my article 😄. I hope you’ve learned a thing or two about gene editing and rare diseases after reading this article. It kinda sucks that we probably won’t get super speed or shooting lasers out of our eyes for a while.
However, helping out people with technology is always a plus in my books. Btw, here’s my contact info if you want to talk (I don’t bite AHAHA):
🔗: Qamil Mirza
Oh, I also have a newsletter covering what exciting stuff I’ve been up to this month so check it out if you want to join me on my journey:
Talk to you later hehe,