Monday, March 27, 2023

What doesn’t kill us can make us stronger: Gene therapy with viruses

There are more viruses in the universe than stars. To get an idea of ​​how big a quantity is, Anyone reading this article would be sitting on top of more than 800 million viruses,

These tiny infectious agents have been with us long before they emerged as a species. And the debate about their presence on Earth is not yet closed: do they predate bacteria, or are they halfway between them and eukaryotic cells?

We don’t know yet, but what we do know is that they’ve been on everyone’s lips during these last two years. It’s nearly impossible to turn on the television, read the newspaper, or walk into a bar without hearing the word “virus.”

medically modified virus

Viruses are very different from each other, and can infect both human cells and bacteria and even plants, causing endless diseases. Who hasn’t caught a cold or the flu and has to spend a few days without getting out of bed?

TookThe history of viruses parallels the history of the development of modern medicine, So much so that nothing has generated such a massive amount of global and unitary effort to achieve protection against them: Vaccination,

In addition, in our recent history, we have achieved another milestone with respect to the virus. Thanks to science, we’re able to turn the tables and a priori turn an evil agent into his nemesis: a drug.

gene therapy

Some are diseases caused by errors in the genome, including cancer. These errors or mutations cause genes within cells to stop functioning, preventing it from doing its job and causing various problems that end in pathology.

Gene therapy is responsible for the treatment of this type of disease. The idea behind this therapeutic strategy is quite simple: if the gene’s function has been eliminated, why not carry a copy of the good, functional gene into the cells? That way they can recover and continue to fulfill their mission.

The question is how to do it. The first obstacle in our journey to the nucleus of the cell, where genetic information is stored, is the plasma membrane, an envelope that maintains and protects all cellular components.

To pass this without harm to our genes, lipid vesicles can be used, which due to their nature are integrated into the membrane, releasing their contents towards the interior. Another possibility is to use an electrical discharge that produces transient pores on the surface of the cell.

However, wouldn’t it be much cheaper to take advantage of mechanisms already in nature? One that does exactly this: introduce genetic material into human cells? We, of course, mention the virus.

Gene Therapy Virus
Gene therapy with viruses: Science has achieved the unimaginable: turning a pathogenic agent into a drug. , Photo: CDC | Unsplash.

virus as vehicle

Viruses are able to infect human cells and introduce their genome into them. They use the host cell’s own machinery to replicate and are thus able to infect nearby cells, until the body’s immune system is able to eliminate this new viral strain.

Thanks to advances in genetic engineering, scientists have been able to modify the genomes of viruses and clip their wings. i.e. transform them so that they can infect but not replicate.

And not only that, but it has also been possible for these agents to carry the genes they want within themselves without waking up the immune system and causing an adverse reaction. In this way it has been achieved that they employ a taxi, a train or a bus carrying passengers (pharmaceuticals) to the destination which one chooses.

retinal dystrophy and butterfly baby

Thanks to these advances, diseases that previously had no cure can be treated, such as hereditary retinal dystrophy, which causes vision loss in children and adults due to mutations in the RPE65 gene. Through an injection, the modified virus that contains a functional copy of this gene will travel to the retinal cells, infect them and deposit the dominant copy inside them.

A more recent example is clinical trials that are being done to treat a range of open wounds Those so-called butterflies appear on the whole body of children. These patients lack the gene for collagen VII and, thanks to the modified type I herpes simplex virus, the functional gene can be carried into skin cells, leading to wound closure.

Currently, the number of diseases that can be treated with gene therapy are counted on the fingers of one hand. However, we need many fingers to count the number of clinical trials being conducted in the hope that one day these treatments may reach hospital wards.

Immune response to treatment, drug specificity and its administration are the main challenges that hinder the progress of these trials and gene therapy approaches to patients.

The simple idea of ​​turning one disease-causing agent into another that cures them has succeeded in restoring function to organs that had forgotten how to function. It has given hope to patients whose medical options were scarce and have been able to improve their living conditions.

With time, effort and financial investment, this drawing rooted in science will help to solve various diseases.


This article was a finalist in the second edition of the youth promotion competition organized by the Lille Foundation and The Conversation Spain.

This article was originally published on The Conversation. read the original.

Nation World News Desk
Nation World News Desk
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