What We Need To Know About Gene Therapy and Genetic Modification

We tell ourselves not to eat foods that contain GMOs. But is gene therapy any different?

The DNA strands in our human genome contain 3 billion base pairs of nucleic acids. A normal gene in a human cell contains 27000 base pairs, though some can go up to be as big as 2 million base pairs.

These genes are used to encode the synthesis of various proteins and enzymes that the body’s cells need to perform their requisite functions.

But of course, defects can occur during replication. If the defect occurred as a result of poor lifestyle choices, we are still able to rectify it by changing up our lifestyle. 

However, if the defect occurred at the stage of the parents’ sperm and egg cells meeting together to form the foetus, self-rectification may not be possible.

In both cases, though, the idea of gene therapy can be involved.

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What is gene therapy?

The United States National Library of Medicine (NLM) defines gene therapy as:

An experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

This concept may sound newfangled to some. Inserting genes already sounds complicated enough.

The whole idea is that the cell may have some defective gene that isn’t producing the requisite amount of proteins (or even worse, producing a malfunctional/misfolded protein that is useless) to be used in the biochemical process that it is involved in.

Therefore, inserting the right gene into the right place should theoretically allow the cell to produce the right protein, and all should go well after that.

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But it ain’t gonna come cheap.

According to the Foundation for Economic Education (FEE),

Unlike other drugs regulated by the Food and Drug Administration, gene therapies are subject not only to the regulatory structure of the FDA, but also to the Office of Biotechnology Activities, and the Recombinant DNA Advisory Committee. Excessive regulatory oversight creates an elongated and expensive route to approval.

As a result, a recently approved gene therapy drug called Zoglensma, which is used for the treatment of spinal muscular atrophy, is going to cost a person US$2.1 million for a single use.

I don’t know about you, but I ain’t got that much liquidity to blow on even a single treatment.

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How does gene therapy work?

From the FEE quote, we can see that gene therapies are regulated partially by the Recombinant DNA Advisory Committee. That gives us a clue in the form of “recombinant DNA”.

In my earlier years as a biomolecular engineering student, the topic of recombinant DNA first came up in the production of biologics drugs (which are much cheaper than Zoglensma, for instance. Humira, a recombinant biologics drug that works on blocking out inflammation receptors, costs approximately US$38000 a year. Not in the range of millions, but still pricey.)

What recombinant DNA technology does is that it takes a plasmid containing nucleic acid base pairs (ranging from a few kilobases to a few hundred kilobases, or the thousands to the hundred thousands) and inserts it into the main DNA strand of the cell.

It is much like jamming a splinter into your finger, to be precise. Just that plasmid insertion is precise — we need to know exactly where to jam that splinter into.

The DNA in the cell must be able to accept and incorporate the plasmid DNA into its overall structure for the gene to be able to express the proteins properly.

The cells undergo a cycle of replication and death. All replicated cells should ideally contain the plasmid DNA fragment as well to ensure that the gene remains functional. Hence, plasmid stability is another key concern. Initial runs with recombinant virus fragments (yes, viruses!) had issues with immune system detection and elimination (oh, autophagy!), hence non-viral plasmid vectors are always more preferred these days.

Precision and stability will contribute significantly to the overall costs of this technology. The analytical technology and the precision skills required of the research team better be good — or else!

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On the topic of genetically modified organisms…

But now we do have to consider if we really do want to use this kind of technology for ourselves.

The thing is… some of us already are.

Plants and animals can be genetically modified with the insertions of plasmids to make them more weather resistant, or to reduce the rate of attacks from vermin.

It’s the exact same concept — plasmid insertion, plant produces new protein, new protein fends off invaders, or new protein provides better resistance to inclement weather…

Every time that we do consume any food that contains genetically modified organisms (GMO), we are already participating in this technology.

Hence, there will be many people who are against the whole idea of consuming GMO foods because it presents itself more of a moral issue than of a scientific issue.

As Professor Sydney Scott said,

“So people might feel extremely about genetically modified food because it’s very unnatural in a way they find almost morally upsetting.”

Therefore, since the technique is one and the same, a person who feels morally upset by the mere thought of GMO food ought to have the same level of misgivings towards gene therapy.

But is that the case? We don’t have any survey data to prove or disprove that notion.

Of course, gene modification in crops and animals can be done more cheaply than gene modification in humans, because the regulations on human life are much stricter than on plant and animal survival!

Where are you on your beliefs towards GMO foods and gene therapy, though?

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