Could the same technology that made several COVID-19 vaccines possible also protect people from cancer or HIV? Possibilities in this area of gene therapy are suddenly wide open thanks to innovations in messenger RNA (mRNA).
Scientists have understood mRNA for years: It carries genetic information in the form of instructions for making vital proteins, making it a vital part of all life. While DNA gets all the attention for holding our genetic blueprints, life could not exist without the work of the proteins made possible by mRNA. And while COVID-19 vaccines have thrust mRNA into the spotlight, the fundamental research into this area has been underway for decades.
One pioneer in mRNA research is Katalin Karikó, an adjunct associate professor at the University of Pennsylvania and a senior vice president of BioNTech, the German company that developed a COVID-19 vaccine with Pfizer. That vaccine and others like it use mRNA technology, but instead of instructing the body to make proteins, it tells the body to make inert fragments of the virus so the immune system can prepare for its threat.
Karikó first proposed using mRNA technology for gene therapy in 1990, and has worked in the field ever since. The broader field of gene therapy, which also includes research into stem cell therapies, will only become more prominent in the future. After all, it’s only natural to see a technology with the capability to turn the tide against a global pandemic and wonder how else it could be applied.
Just look at Moderna: That company is using mRNA technology in both its COVID-19 vaccine and a pair of potential vaccines for HIV, which it will test in a 56-person clinical trial estimated to start later this month. That study, sponsored by the International AIDS Vaccine Initiative, is estimated to last until May 2023 and will be the first time mRNA vaccines against HIV have been used in a human trial. It’s hard to overstate the impact an HIV vaccine could have: The World Health Organization estimates 38 million people currently live with HIV, including 1.5 people who contracted it last year.
That’s not all gene therapy can do: It could potentially be used to treat any number of illnesses or disorders that impact humans, like traumatic brain injuries, eye degeneration, heart disease and blood disorders. In fact, trials for those very subjects are among the more than three dozen gene therapy or stem cell clinical trials underway at UC Davis. Many of those trials are made possible by the UC Davis Institute for Regenerative Cures, where the UC Davis Stem Cell Program and others partner on more than a dozen disease-specific teams and more. That important research is led by Jan Nolta, director of the institute and a professor of cell biology and human anatomy. She’s even working on possible ways to use stem cells to repair parts of the brain damaged by Huntington’s Disease and some forms of Parkinson’s Disease.
“This is such an exciting time in medical science because patients who currently have limited treatment options may soon have opportunities to benefit from stem cell therapy,” she once said at an event dedicated to stem cell therapies.
Whether it’s UC Davis’ technology to potentially treat spina bifida in babies before they’re born or Excision BioTherapeutics’ innovative attempt to use CRISPR technology to create a treatment for HIV, it’s clear these particular areas of biotechnology merit more investment. If you’re researching ways to protect people from these kinds of widespread illnesses or disorders, feel free reach out to me so we can discuss ways to bring help and hope to even more people.
