Panier sees ‘the future is very bright’ for gene therapy | Nebraska today

development of vaccines for Corona virus disease-19 in 2020 was a huge step forward for more than 150 years of genetic research and paved the way for an explosion of medical treatments that could save countless lives.

Angie Banier, Chair of the Swarts Family of Biological Systems Engineering, introduced Nebraska Lecture On November 17, she outlines the history of her field and the challenges and promises ahead.

“It’s an exciting time to be in this field,” said Panier. “It is an exciting time to be a patient whose life may be dramatically changed by these therapies.”

Banier, also a professor of biomedical engineering, has paid tribute to the many giants of science who have advanced genetic research, from Mendel and Morgan to Biddle, Watson, Crick and Franklin, as understanding has grown from identifying the purposes of genes to learning how to manipulate them and deliver them into patients’ cells to cure disease. There have been setbacks along the way, sometimes tragic, that have forced scientists and regulators to regroup and rethink their approaches.

But advances continued in gene therapies to combat diseases including leukemia, a condition that causes blindness, hemophilia, and spinal muscular atrophy in infants.

When reports emerged from China in early 2020 of a new pneumonia-like disease, scientists in the field of vaccines and gene delivery realized that we…we would need a vaccine approach that would allow rapid manufacture and highly effective and efficient outcomes. “The ability to mobilize it quickly and all over the world.”

The traditional approach to developing vaccines has been to take the target virus, inactivate it in some way, and give it back to patients.

“It can take a long time to isolate the virus, grow huge amounts of it and purify it,” Panier said. Which is why it takes months for us to get ready for the flu vaccine, for example.

But there are other ways to achieve this, including using DNA or RNA and its delivery by non-viral means or viral vectors. “And this is where the field really settled in really quickly because we knew we had decades of research in these areas, and we knew these were technologies that were going to allow us to mobilize quickly.”

double stranded DNA Lonely stranded RNA They are the genetic repositories of our cells—the former responsible for storing our genetic information, and the latter for passing it on to the molecules that control our body’s functions, Panier said.

Six out of 11 Corona virus disease– 19 vaccines have been granted emergency approval by the World Health Organization so far DNA or RNA To deliver disease resistance genes.

“It gives me chills to say that this is an area I’ve been working in my entire academic career and more than half of this field Corona virus disease– 19 vaccines that take advantage of technology that in the late 1990s we were worried would never be able to reappear on the market,” Banier said.

Vaccines have been a global success, Panier said, and the future is bright. The effectiveness of gene replacement and vaccines has been proven. There have been hundreds of FDA applications for clinical trials of gene therapy products in the past two years. It is estimated that Food and Drug Administration It will approve 10 to 20 treatments annually by 2025 after about 30 years when only three were approved. New developments in gene-editing technologies are also set to revolutionize cell and gene therapy.

But challenges also remain. The cost is great.

“These drugs cost a lot of money to manufacture,” Panier said. “They cost a lot of money Research and development. Our health care and insurance systems are not well set up to price treatments. And so we have to tackle these economic challenges.”

Pannier’s lab is looking for less expensive ways to deliver these therapies, which can reduce their costs. One possible solution: use extracellular vesicles, which are naturally released from most cell types, function in cell-to-cell communication and deliver cargo from donor to recipient cells.

“We thought if cells were already using this as a delivery method, could we use it as a way to deliver goods DNA And the RNA that we want to offer? “

Panier said this research could eventually lead to oral administration of vaccines, which is a major advance over the needle.

“There are a lot of advantages to giving any drug by mouth: You have high patient compliance. Most people would rather take an oral drug than take an injection. We don’t have medical personnel to administer. We have a lot of space where we can deliver the drug, and we can get a local response or regular.”

Early results so far are promising.

“The future is very bright. I love to tease my students, it’s so cool to suddenly be working in gene therapy again,” Banier said.

The Nebraska Lectures: Chancellor’s Distinguished Speaker Series is presented once per semester, sponsored by the Office of Economic Research and Development, the Chancellor’s Office and Research Council, in collaboration with the Ussher Institute for Lifelong Learning. The Nebraska Lectures bring together the university community with the greater community in Lincoln and beyond to celebrate the intellectual life of the University of Nebraska-Lincoln by demonstrating the college’s excellence in research and creative activity. The topics of these free lectures reflect the diversity of faculty members’ accomplishments in the arts, humanities, social sciences, and physical sciences.

Leave a Comment