Are we on the cusp of a gene editing revolution?
Imagine a world where no child has down syndrome, no one is blind, no one is disabled, and nobody is suffering due to any genetic disorder. That world is closer to us than we think. The future is CRISPR.
CRISPR (Clusters of Regularly Interspaced Short Palindromic Repeats) technology was adapted from the natural defense mechanisms of bacteria and archaea (the domain of single-celled microorganisms). These organisms use RNA and various CaS proteins, including CaS9, to counter attacks by viruses and other foreign bodies. They do so primarily by chopping up and destroying the DNA of a foreign invader. When these are moved into other, more complex organisms, they manipulate their genes, or “edit” them.
Due to its simplicity and efficiency, CRISPR technology might just be the next big thing. It’s a powerful tool for editing genomes and doctors have recently used this gene-editing tool in the human body for the first time.
The test-subjects for this technology used to have Leber Congenital Amaurosis- a condition caused by a gene mutation that prevents the body from making a protein needed to convert light into signals to the brain. Consequently, they were born with little vision and could even become completely blind in the future.
Leber Congenital Amaurosis cannot be treated with standard gene therapy which requires a replacement gene, because the gene needed is too big to fit inside the disabled viruses used to carry it into cells. Researchers intend to edit or delete the mutation through CRISPR by making two cuts on either side of the DNA and hoping that the ends will reconnect and make the gene function as it would on a normal basis.
Gene editing is not something alien to us, so why is CRISPR so special? First, CRISPR technology is cheap and easy compared to other methods that cost thousands of dollars. Also, unlike other gene-editing tools, this technology can be used to target multiple genes simultaneously. Additionally, unlike its counterparts, CRISPR doesn’t need to be paired with separate cleaving enzymes.
These are some of the many advantages of CRISPR. As much as this powerful tool can revolutionize things, we need to be ethical about its use. Power can be misused, but here’s to hoping for a “modified future”.