mRNA vaccines aren't gene therapy, but both are revolutionary scientific innovations

Politicians waging war on science they don't understand will cost us life-saving medical treatments

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What a time to be alive—and a scientist.

Today, we have more knowledge and more tools to improve human health (and the planet) than ever before in human history.

mRNA vaccines and gene therapy are revolutionizing medicine for infectious diseases, autoimmunity, cancers, and genetic disorders. Beyond COVID-19, mRNA vaccines are in development for norovirus, influenza, mpox, Epstein-Barr virus (EBV), and even cancers like pancreatic, small cell lung, and melanoma.

Since reality is nothing if not ironic, some US states and their uninformed politicians, are trying to ban these lifesaving medical interventions.

Not just COVID-19 vaccines. Not just infectious diseases vaccines, either.

Some states have decided that mRNA technology for any vaccine or medicine is a deal-breaker. But wait, there’s more.

Some states have decided that all nucleic acid-based medicines—mRNA vaccines, gene therapies, and even biologic drugs using genetic engineering—must go.

• Montana wants to outlaw all “gene based vaccines” (in their language, includes any mRNA and DNA technologies) for infectious diseases and penalize healthcare providers who administer them. How nice that they have a little carve out for gene therapies for cancer and genetic diseases. Almost like they know this science is revolutionary, but they’re pushing a false narrative for political leverage.

• Iowa wants to ban to administration of any mRNA or DNA-based vaccines in the state and fine anyone who administers them. (they don’t have a carve out for cancer or genetic diseases—yet, but let’s watch the space)

• Florida wants to ban any mRNA vaccine mandates. So, this would apply to school requirements for COVID-19 vaccines and any future vaccines that use mRNA as antigen.

  • If you missed it, preclinical and clinical research for a myriad of mRNA-based vaccines, for infectious diseases like norovirus, influenza, mPox, EBV and many cancers like pancreatic, small cell lung, and melanoma, are underway.

• Idaho isn’t even pretending they care about health. They want to ban all “human gene therapy products” until 2035, including any and all medicines using any form of nucleic acids, which would apply to mRNA (which they call memory RNA), “genetically modified microorganisms,” and other “engineered site-specific nucleases.” (Idaho health department employees aren’t allowed to administer COVID-19 vaccines either.)

A quick question for Idaho politicians: will this apply to insulin? Human insulin is produced by a GMO — the very first FDA-approved GMO, in fact, back in 1982. Since it is produced by transferring a human gene into microorganisms like bacteria and yeast for them to produce in large quantities, it would qualify as a “human gene therapy product,” under their proposed law. Idaho, will you be banning insulin next?

I’ve written at length about the rejection of science by politicians—and across ideologies (read here, here, and here for examples).

But this widespread rollout of anti-science policy will have have disastrous consequences for human health, agriculture and food stability, and medical innovation.

Worse? It’s waging a war on biotechnology. Biotechnology that has measurably improved all of our lives, whether people realize it or not. And it’s being driven by people who have no understanding of science whatsoever.

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Why do people selectively reject science?

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Which brings me to my latest Inside Immunity column for Skeptical Inquirer—AND my first print column. I was honored to be invited to transition some of my pieces to print. Now, every issue of Skeptical Inquirer magazine will feature my column (6 a year).

After writing this back in December (print deadlines are wild) —several states have since introduced legislation attempting to ban mRNA vaccines and gene therapy. It’s also like misinformation fuels policy more than scientific evidence—it’s also why RFK Jr is now the HHS Secretary.

That’s why we need to talk about it; the future of humanity is under attack.

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It’s been five years since SARS-CoV-2 became a global pandemic—and just as importantly, five years since the deployment of the first FDA-approved mRNA-based vaccine. This innovation is rightfully lauded as the next era in biotechnology and novel therapeutic development as evidenced by Katalin Karikó and Drew Weissman’s 2023 Nobel Prize in Medicine (Karikó et al. 2005). Unfortunately, that very same scientific innovation has marked COVID-19 vaccines as a nefarious plot by the government, Big Pharma, and all us brainwashed scientists who worked behind the scenes to study this new virus and develop these lifesaving vaccines.

Disinformation about COVID-19 vaccines echoed familiar false refrains about “harmful ingredients,” dangerous side effects, the supposed inferiority of vaccine-mediated immunity compared to natural immunity, and the purported lack of safety testing. But the advent of mRNA vaccines brought new accusations too, including the claim COVID-19 vaccines were untested “gene therapy.”

Much of the COVID-19 vaccine disinformation was fueled by politicization, with claims that mRNA vaccines were “harmful gene therapy” accelerating because of fundamental gaps in knowledge about molecular biology, colloquial misuse of the term gene even by those with good intentions, and anti-science and medical conspiracism more broadly.

Vaccines Have Evolved over 300 Years—and That’s a Good Thing

Vaccines are tools that train our immune system to recognize and respond to a pathogen without exposing us to the disease-causing microorganism. Vaccines deliver molecules that activate our immune system, called antigens, that lead to the generation of memory immunity. When we encounter the actual pathogen in the future, that immune memory enables us to mount a rapid and effective response, limiting disease severity and preventing death.

Antigens take many forms, but when it comes to infectious diseases, they are commonly proteins or polysaccharides (carbohydrates) displayed on the outside of a microorganism because that location means direct interaction with immune cells in our body.

Vaccine science has evolved substantially from the crude inoculations of live pathogens 300 years ago to the refined formulations we have today (Pollard and Bijker 2021). Through scientific research and development, different formulations of vaccines have been developed, each balancing the ability to elicit that immune response with the feasibility of manufacture. Today, vaccine technologies include:

• Attenuated vaccines (using a weakened form of the pathogen)

• Inactivated vaccines (using a killed pathogen)

• Subunit vaccines (using only specific antigenic components of the pathogen)

• Toxoid vaccines (using inactivated toxins from the pathogen)

We can now add mRNA vaccines, the newest chapter in vaccinology, to that list. mRNA isn’t an antigen directly but rather the blueprint for our cells to produce antigen themselves.

What Is mRNA Anyway?

A gene is a segment of a DNA molecule that has multiple regions, including a promoter, exons (the bits that dictate sequence of amino acids in a protein), introns, and terminator regions. Genes are blueprints for making proteins or other functional molecules. Every chromosome we have (and other eukaryotic organisms) contains many genes. For eukaryotes, all our DNA—all our genes—lives in the nucleus of each of our cells.

To send the messages for which genes need to be expressed, DNA must be converted into something that can leave the nucleus. Enter mRNA, short for messenger RNA, a molecule identified in 1961. mRNA is not a gene; it is a transcript. That is, mRNA is a temporary copy of a specific DNA sequence that is transported out of the nucleus and designated to be used by our ribosomes, which converts it into a functional protein (Crick 1970). mRNA is a short-lived intermediate that is used as a message relay signal. After it is read by ribosomes and used to string up a chain of amino acids to become a protein, it rapidly degrades.

COVID-19 mRNA vaccines use an mRNA that is the template for the spike protein of SARS-CoV-2, a protein on the virus that serves as an antigen. Unlike a vaccine directly delivering manufactured spike protein, mRNA vaccines merely contain the blueprint for the spike protein. Upon vaccination, our cells take up the mRNA, produce spike protein, and present it to the immune system. This causes the immune system to flag that protein as foreign and generate memory immunity—without ever exposing the body to any significant components of the virus itself (Rando et al. 2023; Pardi et al. 2020).

mRNA is one degree removed from the antigen our immune system would recognize, but because our cells abide by the central dogma of molecular biology, they read that mRNA segment and synthesize protein from it. This technology is incredibly elegant and has been in research and development for over thirty years (Dolgin 2021).

While many people cast doubt on mRNA vaccines because they are a newer vaccine technology than others, it should be viewed as what it is: the advancement of science with increasing knowledge. If we all took the attitude of dismissing newer—and improved—technologies, we would still be snorting dried smallpox scabs up our noses (Needham 1980; Boylston 2012)!

mRNA Vaccines Aren’t Gene Therapy because mRNA Isn’t a Gene

The misconception that mRNA vaccines are “gene therapy” comes from a combination of factors, but I would say there are two main drivers.

The first exploits a fundamental lack of knowledge about molecular biology by people who are intentionally trying to undermine COVID-19 vaccines. These people repeat claims that suggest these vaccines—and gene therapy—are tactics to control the population, to “alter our genetics,” and to make people transhuman. While none of these has any basis in reality, the goal is to erode trust in scientific institutions and federal agencies, a common ploy of those who propagate medical conspiracism (Oliver and Wood 2014).

The second resulted from colloquial misapplication of the word gene, even coming from well-intentioned scientists trying to simplify complicated science at a time when people were bombarded with clickbait headlines, uncertainty, and disinformation.

In biomedical science, we often casually refer to anything related to genetic material as genes, even when it isn’t scientifically accurate. It’s a shorthand way of referring to a gene product, because those of us in the field have a deep understanding that a gene transcript (the mRNA) originates from the gene itself (the DNA). Unfortunately, if this critical distinction isn’t explained properly, it can mean even an innocent statement (simplified to limit confusion with even more molecular biology) gets conflated and weaponized. People looking to spread disinformation about vaccines will grasp anything they can to drive misinformation campaigns—and that’s even more true when concepts about genetics and genes come into play.

Gene Therapy Isn’t Something to Fear Either

Gene therapy is a catchall term for therapeutic interventions that aim to alter, repair, or replace a gene (DNA) to treat or cure diseases that result from defective genes. Gene therapies involve modifying, silencing, or replacing DNA segments, which reside in the nucleus of target cells.

mRNA vaccines don’t do this. mRNA doesn’t enter the nucleus of the cell, where our genes reside. Our cells are compartmentalized and cellular functions have directionality, so mRNA in a vaccine doesn’t travel into the nucleus of a cell where it could interact with our genes. mRNA, whether in a vaccine or produced naturally by our cells, is a temporary instruction for ribosomes to produce proteins.

Gene Therapy Is a Revolutionary Biotechnology That Can Treat the Untreatable

During the COVID-19 pandemic, anti-vaccine activists painted mRNA vaccines as gene therapy to insinuate they were harmful. Well, COVID-19 vaccines aren’t harmful; they are estimated to have saved tens of millions of lives globally. Neither is gene therapy harmful. They just aren’t one and the same.

Gene therapy is a life-changing therapeutic field that is allowing us to treat diseases previously considered incurable. Gene therapies can provide long-term or permanent corrections to genes that cause chronic or fatal diseases. Many of these diseases are inherited, have no therapeutic options, and current treatments merely alleviate symptoms or modestly slow disease progression. Gene therapies can deliver the corrected gene directly, or we can alter genes within certain cells to combat a disease.

The very first FDA-approved gene therapy was in August 2017 for treatment of B-cell acute lymphoblastic leukemia (B-ALL). Kymriah (tisagenlecleucel) is a gene therapy that introduces a gene into T cells to produce a protein that binds an antigen on B cells (which are cancerous in B-ALL). These gene-modified cells, CAR (chimeric antigen receptor) T cells, can now target and destroy those B cells, treating the leukemia (Maude et al. 2018).

That same year, the first gene therapy for a genetic disease was also FDA-approved. Luxturna (voretigene neparvovec-rzyl) was approved in December 2017 for inherited pediatric blindness due to RPE65-associated retinal dystrophy.

I’ll give you one guess as to which gene is involved! That’s right, RPE65. RPE65 produces an enzyme, retinoid isomerohydrolase, that converts vitamin A into retinal, which is essential for detecting light in retinal cells. With a defective RPE65 gene, you don’t produce the enzyme, the conversion doesn’t occur and light isn’t detected. This means you can’t see, and that vision loss worsens over time.

Luxturna replaces that defective RPE65 gene with a functioning one through a subretinal injection (yep, in the eyeball). When the functioning RPE65 gene is delivered, your eye cells can produce the enzyme, and vision can be restored (Russell et al. 2017).

Pretty incredible, right?

In the seven years since, around forty gene therapies have been FDA approved for an array of diseases from rare pediatric neurodegenerative disorders, pediatric blindness, cancers, and hematologic diseases. We have treatments for conditions such as spinal muscular atrophy (SMA), sickle cell disease, hemophilia A, beta-thalassemia, Duchenne muscular dystrophy (DMD), multiple myeloma, B cell lymphoma, and more.

Many genetic diseases are not only rare but fatal. Because they’re frequently inherited, they often disproportionately impact children or certain ethnicities. Gene therapy is a new era in therapies for conditions that were once untreatable, many of which impact marginalized populations.

While the term gene therapy may connote fear, this is often because of misunderstanding about the science and disinformation that started in the 1990s, when claims alleged gene therapy would “rewrite your entire genome.” The reality is these treatments are rigorously studied, monitored, and are targeted specifically to the defective gene.

mRNA Vaccines and Gene Therapy Are Biotechnology Advances That Should Be Celebrated

mRNA vaccines and gene therapies are both incredible advancements in science and medicine. Their development and therapeutic successes underscore the scientific method: harnessing the continued accrual of knowledge and technology to innovate and improve health outcomes. The COVID-19 pandemic illustrated the potential of mRNA vaccines. Clinical trials are underway for cancers, HIV, malaria, and more. Gene therapy research and development continue to target currently incurable diseases such as cystic fibrosis, muscular dystrophy, and more.

While mRNA vaccines are not gene therapy, fear around both classes of therapeutic innovations is rooted in misinformation, not science. Both represent scientific developments that are the result of embracing progress. These tools can shape the future of medicine—and human health—so long as we don’t let misinformation get in the way.

References can be found here.

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Now, more than ever, we all must join in the fight for science.

Thank you for supporting evidence-based science communication. With outbreaks of preventable diseases, refusal of evidence-based medical interventions, propagation of pseudoscience by prominent public “personalities”, it’s needed now more than ever.

More science education, less disinformation.

- Andrea

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ImmunoLogic is written by Dr. Andrea Love, PhD - immunologist and microbiologist. She works full-time in life sciences biotech and has had a lifelong passion for closing the science literacy gap and combating pseudoscience and health misinformation as far back as her childhood. This newsletter and her science communication on her social media pages are born from that passion. Follow on Instagram, Threads, Twitter, and Facebook, or support the newsletter by subscribing below: