Can mRNA therapy cure other diseases?

Can mRNA therapy cure other diseases?  The mRNA vaccine competition against COVID-19 does not solve the administration problem faced by mRNA therapy, but it may pave the way for mRNA therapy in other ways.

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In November last year, two new coronavirus pneumonia vaccines achieved great success in clinical trials, and the U.S. Food and Drug Administration (FDA) subsequently approved the mRNA vaccine jointly developed by Pfizer and BioNTech and the mRNA vaccine developed by Moderna for emergency use . In fact, long before the COVID-19 pandemic, mRNA vaccines have attracted a large number of pharmaceutical companies to participate in research and development with their unique advantages.

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Messenger RNA (mRNA) is a nucleic acid molecule that transfers genetic code from DNA to protein-making machinery. Scientists hope to use it as a translation template to redesign a set of multifunctional drugs or vaccines to produce therapeutics in the body. Or anything needed to prevent disease.

The mRNA vaccine is to directly introduce the mRNA encoding a certain viral antigen protein into the animal’s somatic cells, and synthesize the corresponding antigen protein through the host cell’s translation system, thereby inducing the host to produce an immune response to the antigen protein to achieve prevention and The purpose of treating viral infections.

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The biggest challenge in the clinical application of mRNA therapy is how to target mRNA to specific tissues and provide strong and long-lasting therapeutic effects without excessive side effects.
In fact, instead of putting in an mRNA sequence, it can treat any disease you want to treat. Tailoring an mRNA drug for a certain disease usually means adjusting both the mRNA itself and the structure of lipid nanoparticles used for transportation in the body.

Lipid Nanoparticles (LNP) for the treatment of tumors　

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Of course, for some mRNA therapies, the way of administration is relatively simple. For example, mRNA vaccines are usually injected intramuscularly into the vaccinators. Muscle cells obtain mRNA and produce corresponding viral proteins. The host immune system is stimulated by these viral proteins to produce antibodies and T cells to resist future invasions.

Other local injection methods (such as subcutaneous injection or tumor injection) can also provide some mRNA-based treatments that use the immune system to fight cancer. These therapies can help enhance the body’s attack on cancer cells by encoding tumor proteins or immune signaling molecules. In more cases, mRNA drugs are still delivered through lipid nanoparticles (LNP). It is worth noting that sometimes the wrong delivery of mRNA drugs can lead to serious consequences.

For example, in mRNA therapy to treat ornithine carbamate (OTC) deficiency, the enzyme encoded by the mRNA can cause the accumulation of ammonia in the blood, which can induce epilepsy, coma and even death. Therefore, many pharmaceutical companies are working to reduce or eliminate the possible defects of this mRNA therapy-by adjusting the structure of lipid nanoparticles, or modifying them with molecules to allow them to enter specific organs or cell types.

Today, the mRNA vaccine race against COVID-19 has not solved the delivery problem faced by mRNA therapy, but it may pave the way for mRNA therapy in other ways. Nowadays, many pharmaceutical companies and laboratories around the world are trying to find the best lipid nanoparticles (LNP) to deliver mRNA safely and efficiently to specific locations in the human body. I believe this transformative day will finally come.

(source:internet, reference only)

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