CRISPR Genome Editing: What Does It Mean For Stem Cell Treatments?

17/06/2016

Being able to cut and paste away at the biological code of life is the stuff of science fiction.

Genetically modified crops, designer babies, animal cloning; these are just a few examples of how modern day medicine is already playing the role of God.

But with something so incredibly complex as our gene editing, there’s still a lot we don’t fully understand.

One wrong edit in the DNA of a crop could potentially disrupt a whole ecosystem, just as one change in a human’s DNA could cause unexpected harmful effects.

But the potential advantages are unignorable. From producing disease resistant crops to curing cancer, gene editing has the potential to revolutionise medicine and change humanity in unimaginable ways.

So what if there was a way to edit genes that was not only safe and easy, but also fast and cheap?

Well, with CRISPR, all this and more becomes possible.

CRISPR: A Natural Gene Snippet System Found In a Surprising Place

It turns out gene editing isn’t something we invented, but rather a biological mechanism that’s been around since the dawn of all life.

CRISPR, short for clustered regularly interspaced short palindromic repeats, is a natural system—first identified in E. coli bacteria, that’s essentially a database of DNA collected from external threats.

When first discovered in 1987, scientists were confused as to why the DNA of the bacteria had DNA from other sources inside it. But since then, they’ve realised it’s part of the bacteria’s immune system—storing snippets of DNA so it learns what is harmful and what it needs to respond to.

What’s so remarkable about this discovery is that the bacteria works with a tool  known as CRISPR-cas9, which, when invaded by a virus, snips the piece of DNA and delivers it to the database for storage.

This natural editing tool was first recognised by Jennifer Doudna (check out her TED talk), as a potential way to modify other genes. She suggested if bacteria were fed a segment of the target DNA that’s known to be bad—for instance a gene that causes blindness—it would store it in its CRISPR database, so the bacteria could then be introduced to the body to seek out and assassinate the bad gene.

Doudna was right, and in the past four years the CRISPR system has been used to reverse blindness, halt cancer cells from multiplying, treat HIV, help create disease-resistant crops like rice and wheat, along with countless other advances.

A Perfect Compliment To Stem Cell Therapy

Stem cells are the body’s ‘mother’ cells, from which every other cell, tissue, and organ arises from.

Being able to edit our stem cells using CRISPR opens up many applications for therapeutic treatments, and holds the promise of curing thousands of genetic diseases—one example being muscular dystrophy, which affects hundreds of children each year.

A thanks to the natural mechanism behind CRISPR-cas9, gene editing can be done with much precision and ease than ever before.

One promising method in which CRISPR could be used is in conjunction with stem cell transplants.

The problem with treating diseases such as cancer with stem cell therapy is that chemotherapy or radiotherapy is still needed first to kill off diseased cells. But using the CRISPR-cas9 system, bacteria could be introduced to the body to find and eliminate the diseased cells—avoiding harmful drugs which also kill your body’s cells—before healthy stem cells are transplanted back into the blood.

What’s more, if someone is suffering from leukaemia, it’s unlikely their own stem cells could be used for the treatment due to the nature of the disease. Cutting the bad gene from their faulty stem cells using CRISPR could provide a solution.

Treatments using CRISPR may not be as far away as you think, with companies such as Intellia Therapeutics, Editas Medicine, and CRISPR Therapeutics, all in the race to pioneer these exiting advancements in gene editing and healthcare and introduce them to the public.

CRISPR is opening up many new doors for stem cell therapy. Visit our website and find out how you can store the stem cells from your children’s milk teeth.