CRISPR coming of age

 
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Since the first mapping of the human genome in 2003, the understanding and development within genomics has moved quickly. When the CRISPR Cas9 system was discovered in 2013 (a fairly simple and low-cost method to alter any gene in any organism) things went ballistic. Now, we have a technology that can potentially eradicate most genetic diseases. It could also change an entire animal species or alter an embryo to decide gender or eye colour.

In 2019, the world’s first gene-edited twin girls were born in Shenzhen, China. This action was broadly condemned, but clearly shows what is possible. We already have some of the first FDA-approved treatments for genetic diseases – Luxturna from Spark Therapeutics, for example, treats a specific type of inherited retinal dystrophy that stems from mutations in a gene known as RPE65 (the treatment is priced at $850.000 per patient). Zolgensma from Novartis promises to treat Spinal Muscular Atrophy, a rare, genetic, neuromuscular disease, with just one injection. This treatment currently costs $ 2.1 million, leading to debates about pricing and benefits. Who decides the cost of a life?

While this discussion heats up, several new and exciting techniques are being developed – if CRISPR-Cas9 works like a scissor and a pencil to cut and paste new information into your DNA, then a new technique called “Prime editing” works more like a word processor, making almost any alteration possible — additions, deletions, swapping any single letter for any other, without severing the DNA double helix.

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Gene Drive is another genetic engineering technology that can spread particular genes across a population. This could potentially eradicate malaria-transmitting mosquitoes, but also risks unintended consequences. Clearly, there is room for more discussion on ethics. What chances are we willing to take? Who should be allowed to perform these alterations and in what situations?

Meanwhile, anyone can log on to www.the-odin.com and purchase a DIY Bacterial Gene Engineering beginners CRISPR Kit for just $159 (which many nerds have done). This has led to communities of so-called Biohackers across the world, like Syntech Bio network – an online community of more than 4000 science lovers and tinkerers. It is clear that this technology is extremely hard to contain within set boundaries.

I predict several major commercial breakthroughs within gene-editing in 2020, as well as heated debate on the subject. If you want to dig deeper into this topic, I strongly recommend the Netflix documentary series “Unnatural Selection”, released in October of 2019.