CRISPR 101-Manipulating our DNA

“DNA, the molecule of life”

As living things, not just humans, but all living things, we are made up of DNA. DNA sequences determine physically, who we are. It determines our eye colour, our hair colour, our likeliness to develop certain diseases, lactose levels and much much more.

Now changing these genes, that’s another story. It sounds like something straight out of a movie. But guess what? It isn’t. Our technology has advanced so much, that editing our genes is no longer a fantasy.

What Is Gene Editing?

In simple words, gene editing is a group of technology that allows us to change an organism's DNA 🧬. These technologies allow genetic material to be removed, added or altered at specific locations in the genome.

Yea, I know, this sounds crazy complicated, but thanks to CRISPR it’s become a whole lot easier.

What’s CRISPR?

CRISPR is currently the easiest and most cost-efficient💰 gene-editing tool. There are two main parts to this process, CRISPR itself and another tool by the name of CAS-9.

1. CRISPR already exists inside our bodies. It’s a family of DNA sequences found in the genomes of prokaryotic organisms like bacteria and archaea. CRISPRs are a part of our bacterial immune system and they are of repeating sequences of genetic code, interrupted by “spacer” sequences — a remainder of genetic code from past invaders. The system is essentially a genetic memory to help cells detect and destroy and invaders if they do return. Scientists found a way to engineer these to edit the genome in human cells
2. CAS-9 is an enzyme that essentially works like a pair of scissors but for genetic material and DNA. It can cut the two strands of DNA at a specific location in the genome so that bits of DNA can then be added or removed.

The process begins with RNA, a molecule that can read the genetic information in DNA, therefore it acts as a guide. The RNA finds the spot in the nucleus of a cell (where most genetic material is stored) where the editing activity should take place. The RNA then guides the Cas9 to the spot needed to be cut on the DNA. Cas9 then snips the DNA to create a break in both strands of the DNA molecule.

There are a few different processes to disrupt, delete or insert genes:

• Disrupt: When a single cut is made, it will result in the addition or deletion of base pairs which disrupts the original DNA sequence and causes the gene inactivation.
• Delete: If we use two guide RNAs to target separate sites, we can delete a larger fragment of DNA.
• Insert: We can add a DNA template alongside CRISPR & Cas9 which lets the cell correct a gene or insert a new gene.

Applications Of CRISPR:

This technology is not just a cool fantasy, it will have a huge impact on our world. There are so many potential applications of gene editing, and CRISPR specifically and a large variety too.

1. Cell & Gene Therapies: CRISPR will have the most impact on the medical industry, specifically with curing a huge range of genetic diseases including blood disorders, cancer, ocular disorders, neurodegenerative disorders and more!
2. Diagnostics: We can use Cas9’s search function to detect genetic material from a virus whether it be infectious or genetic.
3. Agriculture: We can also use CRISPR as a tool to create genetically modified foods within the next 5–10 years. This can be used to create disease and drought-resistant crops. It can also be used to prolong the shelf life of perishable foods which will reduce food waste!

Companies To Check-Out:

• CRISPR Therapeutics: Developing transformative gene-based medicines for serious diseases with CRISPR/Cas9
• Regeneron Pharmaceuticals: Biotechnology company, which engages in the discovery, invention, development, manufacture, and commercialization of medicines
• Intellia Therapeutics: Treating ocular diseases with CRISPR/Cas-9
• Editas Medicine: Their goal is to discover, develop, manufacture, and commercialize transformative, durable genomic medicines for many diseases.
• Beam Therapeutics: Beam is pioneering the use of base editing — a potential new class of precision genetic medicines

CRISPR/Cas-9 and gene editing as a whole is no longer a fantasy, it’s here. But it’s up to us as a society to use it effectively and safely to create a safer world where we have effective treatments for every disease, solutions for food waste, and much much more!